Meds Pipeline Monitor 2022

Contact Information

Patented Medicine Prices Review Board
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Email: PMPRB.Information-Renseignements.CEPMB@pmprb-cepmb.gc.ca

Acknowledgements

This report was prepared by the Patented Medicine Prices Review Board (PMPRB) as part of the National Prescription Drug Utilization Information System (NPDUIS) initiative.

The PMPRB wishes to acknowledge the members of the NPDUIS Advisory Committee for their expert oversight and guidance in the preparation of this report. Please note that the statements and findings for this report do not necessarily reflect those of the members or their organizations.

We gratefully acknowledge Patricia Carruthers-Czyzewski, BScPhm, MSc, Sintera Inc. for providing pharmaceutical expertise and for her contribution to the scientific analysis.

Appreciation goes to Allison Carey for leading this project, and Tanya Potashnik and Kevin Pothier for their oversight in the development of the report. The PMPRB also wishes to acknowledge the contribution of the editorial staff, Shirin Paynter.

Disclaimer

NPDUIS operates independently of the regulatory activities of the Board of the PMPRB. The research priorities, data, statements, and opinions expressed or reflected in NPDUIS reports do not represent the position of the PMPRB with respect to any regulatory matter. NPDUIS reports do not contain information that is confidential or privileged under sections 87 and 88 of the Patent Act, and the mention of a medicine in an NPDUIS report is not and should not be understood as an admission or denial that the medicine is subject to filings under sections 80, 81, or 82 of the Patent Act or that its price is or is not excessive under section 85 of the Patent Act.

Although this information is based in part on data obtained under license from GlobalData and the MIDAS® Database proprietary to IQVIA Solutions Canada Inc. and/or its affiliates (“IQVIA”), the statements, findings, conclusions, views, and opinions expressed in this report are exclusively those of the PMPRB and are not attributable to either GlobalData or IQVIA.

Suggested Citation

Patented Medicine Prices Review Board. (2023). Meds Pipeline Monitor, 2022. Ottawa: PMPRB.

Executive Summary

Meds Pipeline Monitor (MPM) is a horizon scanning report that features a selection of new medicines undergoing clinical evaluation or in pre-registration that may have an impact on future clinical practice and drug spending in Canada.

This edition expands the review for the selected medicine candidates in Phase III clinical trials or pre-registration to include information on other drugs in Phase II that share the same mechanism of action or indication. Having insight into other drugs under investigation (i.e., in Phase II) may provide additional information on the potential place in therapy for these pipeline candidates. Medicines in Phase III clinical trials or pre-registration are selected as candidates for the ‘new medicines’ list if they have the potential to address an unmet therapeutic need, offer a novel mechanism of action or therapeutic benefit over existing therapies, or treat a serious condition. The medicines in Phase II are also examined to identify other drugs that are in earlier phases of the pipeline that contain the same indication or mechanism of action as the selected medicine candidates.

The report collects data from two main sources: GlobalData’s Healthcare database, which identifies medicines currently undergoing clinical evaluation, and Health Canada’s Drug and Health Product Submissions Under Review Lists, which provide information on new medicines under review in Canada.

Highlights of the Meds Pipeline Monitor 2022

  • In 2022, the pipeline contained over 9,000 new medicines in various stages of clinical development, compared to just under 8,500 the year before. The number of drugs in the pipeline is increasing by an average of 11% per year since 2018.
  • Oncology continues to dominate the therapeutic mix in 2022, with cancer treatments representing almost one third (30%) of medicines in all phases of clinical trials. Treatments for infectious diseases held the second largest share of the pipeline, at 15%, due to the increased number of medicines for the treatment of COVID-19.
  • Nearly one third (31%) of medicines in Phase III clinical trials or pre-registration had an early orphan designation approved through the US FDA or the EMA, which is consistent with the increasing trend in the prevalence of orphan-designated medicines entering the pharmaceutical market.
  • Trends in the 2022 pipeline include a growing number of novel gene and cell therapies that are expanding to larger patient groups (e.g., Duchenne muscular dystrophy). The biosimilars pipeline is also expanding to therapeutic areas including asthma (omalizumab), bone health (denosumab), and myocardial infarction (tenecteplase).
  • Twenty-eight new medicines including five new gene and cell therapies were selected for the 2022 MPM new medicines list based on their potential to impact the Canadian healthcare system. Nine of the medicines listed in this year’s report have forecasted global annual revenues of over US $1 billion by 2028, one of which was approved by Health Canada in February 2023.
  • Of the 42 new and retained medicines listed in the previous edition (MPM 2021), six received market authorization, 25 were retained on this year’s list as they continued to satisfy the selection criteria, and 11 were removed as their clinical trials were discontinued or they no longer meet the selection criteria.
  • As of September 2022, 550 vaccines and therapies were undergoing clinical evaluation globally for the prevention and treatment of COVID-19. Health Canada is reviewing 14 new and supplemental drug submissions for the prevention and treatment of COVID-19.

List of Terms

For the purpose of this report, the following terms and associated definitions apply.

Cell therapy
The transplantation of human cells to replace or repair damaged tissue and/or cells.
Clinical efficacy
The maximum response achievable from a medicine in research settings and the capacity for sufficient therapeutic effect in clinical settings.Footnote i
Gene therapy
A technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene, as defined by Health Canada.Footnote ii
Market authorization
The process of approval for a medicine to be marketed in a given country. In Canada, market approval is granted following a substantive scientific evaluation of a product's safety, efficacy, and quality, as required by the Food and Drugs Act and Regulations.Footnote iii
Medicinal ingredient
A chemical or biological substance responsible for the claimed pharmacologic effect of a drug product. Sometimes referred to as a molecule, active substance, or active ingredient.Footnote iv
Medicine
A broad term encompassing both the final drug product and medicinal ingredient(s); this encompasses chemically manufactured active substances and biologics, including gene therapies. Medicines are reported at the medicinal ingredient level and can refer to a single ingredient or a unique combination of ingredients.
Patent evergreening
The acquisition of additional patents for minor modifications to an existing pharmaceutical product in order to extend the patent life of the medicine (e.g., new delivery systems, new dosages, new uses, new combinations or new forms).Footnote v
New medicine
A medicinal ingredient that has not previously received market authorization by a regulator.Footnote iv
Orphan medicine
A medicine used to treat a rare disease. For the purposes of this study, orphan medicines are defined as having an orphan designation granted by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for the relevant indication.

Phases of clinical trials

Phase I
These trials test an experimental medicine on a small group of people for the first time. The purpose is to look at the medicine's safety, determine a safe dosage range, and monitor if there are any side effects.
Phase II
In this phase, the medicine is given to a larger group of people (usually 100 or more) to gather data on how well the medicine works to treat a disease or condition, check its safety on a wider range of people, and determine the best dose.Footnote vi
Phase III

These controlled or uncontrolled trials are conducted after preliminary evidence suggesting efficacy of the medicine has been demonstrated. They are intended to gather additional and confirmatory information about the clinical efficacy and safety of the medicine under the proposed conditions of use.Footnote ii Phase III trials are usually randomized with double-blind testing in several hundred to several thousand patients.

Pre-registration
A medicine is in the pre-registration phase once all the necessary clinical trials have been completed and it is waiting for registration or approval for use by a governing body.Footnote vi

Introduction

This edition of the Meds Pipeline Monitor (MPM) features a selection of new medicines in Phase III clinical trials or pre-registration that have the potential to impact clinical practice and drug spending in Canada.

The methodology, which is detailed in the next section, uses a specific set of criteria to identify a list of new medicines in the pipeline from the GlobalData Healthcare database, as well as a list of medicines currently under review from Health Canada’s Drug and Health Product Submissions Under Review (SUR) Lists. The new medicines listed in this report are selected based on a scientific review of the literature and clinical trial outcomes to determine if the medicine may impact the Canadian healthcare system by: addressing an unmet therapeutic need; offering a novel mechanism of action or therapeutic benefit over existing therapies; or treating a serious condition. Medicines reported in previous editions of the MPM are also reviewed and updated in this report. This report also provides an update on the medicines in last year’s edition that have since received market authorization by either the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), or Health Canada. Likewise, the new medicines featured in this report will be monitored in future editions of the MPMto identify candidates that successfully enter the market.

To provide context for the selection of medicines, the MPM includes a snapshot of the number of drugs in each clinical phase of the pipeline year over year (2018-2022), and a breakdown of the various therapeutic areas for each phase of clinical development. This edition of the report also highlights select vaccines and other medicines undergoing clinical trials for the prevention and treatment of COVID-19, in global markets as well as in Canada. The medicines assessed for this portion of the analysis include new therapies as well as previously marketed treatments for other indications that have been repurposed for the treatment of COVID-19.

Meds Pipeline Monitor is a companion publication to Meds Entry Watch, which analyzes the market launch patterns of newly approved medicines in Canada and internationally. Together, these two PMPRB reports monitor the market continuum of late-stage pipeline medicines and new approvals, providing decision makers, researchers, patients, clinicians, and other stakeholders with information on the emerging medicines and evolving cost pressures.

Methodology

Snapshot of the Pipeline

The snapshot of the 2022 pipeline identifies the composition of medicines in various phases of clinical development. For the purpose of this analysis, a full list of pipeline medicines was retrieved from GlobalData’s Healthcare database in September 2022 and the selected medicine candidates for this year’s report have been validated as of March 30, 2023.

New medicinal ingredients are identified as those with no prior approvals through the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), or Health Canada. The distribution of new medicines by therapeutic area corresponds to the indication under evaluation, as reported by GlobalData. Note that a single new medicine may be undergoing multiple clinical studies for separate indications.

Meds Pipeline Monitor

The MPM selects new medicines in Phase III clinical trials or pre-registration in Canada, the United States, and Europe. Many of the pipeline candidates are first-in-class or represent novel mechanisms for treatment in a specific therapeutic area. For this reason, this report includes additional review on other drugs undergoing Phase II clinical evaluation that share the same indication or mechanism of action. Pipeline medicines are selected for inclusion using a two-stage process (Figure 1). The initial screening stage selects medicines in the late phases of clinical evaluation, while the analytic review stage involves a more rigorous appraisal of each potential candidate to identify medicines that may have a significant clinical and budgetary impact.

Figure 1. Selection process for medicines featured in the MPM Figure 1. Selection process for medicines featured in the Meds Pipeline Monitor

* In pre-registration with the US Food and Drug Administration (FDA).
Has Phase III clinical trials in Canada, the United States, or geographic Europe (excluding Russia and Turkey).

Figure description

This is a flowchart describing the process used to select the listed medicines. The chart consists of two steps:

1. Initial Screening

This step begins with all medicines in Phase III clinical trials or pre-registration with the US Food and Drug Administration. Of these medicines, the next step includes only those with expected clinical trial end dates within three years of the analysis and drug geography including Canada, the US, and Europe. To qualify for the drug geography, a medicine must have Phase III clinical trials in Canada, the US, and/or geographic Europe (excluding Russia and Turkey).

2. Analytic Review

The analytic review step of the process is divided into two parts: one path for new medicines and the other for gene therapies.

New medicines must meet at least one of the following requirements to be included in the list:

  • Demonstrates improved safety and efficacy
  • Novel mechanism and/or first-in-class, with the addition of one or more of Breakthrough, Fast Track, and Priority Review designations

Gene therapies must demonstrate clinical effectiveness with an acceptable safety profile to be included in the list.

Stage 1. Initial screening

GlobalData’s Healthcare database is used to identify a list of medicines undergoing Phase III clinical trials or in pre-registration. These medicines serve as the basis for the initial screening stage.

The drug geography, defined as the geographical region or country in which the medicine is either marketed or in pipeline development, is restricted to Canada and other countries with similar regulatory and approval processes: the US and geographic Europe (excluding Russia and Turkey). Only new medicinal ingredients that have adequate data that supports increased efficacy and safety from clinical trials are considered as candidates for inclusion.

Medicines approved or sold in Canada, the US, or Europe for any other indication or in any other strength or formulation are excluded during the selection process, as are medicines whose clinical trials are inactive, suspended, withdrawn, or terminated.

Stage 2: Analytic screening

Selection criteria

Following the initial screening, the second stage of the process considers a number of selection criteria to determine the final list of pipeline candidates. These criteria are detailed in Table 1.

Earlier phases of the pipeline (i.e., Phase II) are also examined to determine if there are other medicines with the same indication or mechanism of action as the selected candidates in Phase III and pre-registration. This provides additional information on the number of novel, first-in-class medicines that are undergoing clinical evaluation in Phase II that may influence the therapeutic significance of the selected candidates in Phase III and pre-registration.

Table 1. Selection criteria for the MPM

Selection criteria
Improved safety and efficacy shown in clinical trials: a medicine that demonstrates increased safety, new outcome measures, or increased life expectancy or quality of life

Novel mechanism / First-in-class: a medicine that uses a new mechanism of biochemical interaction to produce a medical effect, or a medicine that is the first in its therapeutic class

In addition, the medicine must fall into one or more of the three following FDA designations for expedited development and review:
  • Breakthrough – medicines intended to treat a serious condition and for which preliminary clinical evidence indicates that they may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)
  • Fast Track – medicines used to treat serious conditions and fill an unmet medical need
  • Priority Review – medicines that would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications
Gene or cell therapy: a technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene; or the transplantation of human cells to replace or repair damaged tissue and/or cells
Additional descriptive information

A profile of each successful pipeline candidate is provided, including the indication and mechanism of action, as well as a summary of the applicable published outcomes from clinical trials. Specific attributes that may influence the potential uptake or cost of each medicine are also identified. Table 2 provides a detailed description of these key attributes.

Table 2. Key attributes of new medicines selected for the MPM

Attribute Relevance Data sources
Clinical trials in Canada

Medicines tested in Canada are likely to be of interest to Canadians

GlobalData Healthcare;

Health Canada Clinical Trials Database; Health Canada Drug and Health Product Submissions Under Review; National Institutes of Health (NIH) Clinical Trial Registry
Rare or orphan designation Medicines used to treat rare diseases or conditions that generally have high treatment costs and may result in substantial spending GlobalData Healthcare
Biologic medicine These complex molecules produced by living organisms are expected to have high costs, resulting in substantial spending
Add-on therapy Medicines designed to be used in conjunction with existing medicines may increase the treatment cost and contribute to higher spending
Potential evergreening Modified forms of the same product in order to extend the patent life. (e.g., new delivery systems, new dosages, new uses, new combinations or new forms)

The profile also provides details of potential cost implications, if available, which includes the forecasted global revenues reported by GlobalData.

The indications and therapeutic areas of the featured medicines correspond to their Phase III clinical trial or pre-registration stage. A single clinical trial may assess multiple indications within the same therapeutic area. These medicines may also have additional indications at various phases of clinical evaluation that are not mentioned in this report. The scientific description and key attributes provided are focused on the specified indication(s) for the selected medicines.

Medicines reported for a given year are reassessed for each following edition of the MPM. They may be retained on the MPM list if they continue to meet the selection criteria. Medicines for which clinical trials have been discontinued or for which the selection criteria is no longer met are not reported in subsequent editions.

Spotlight on Canada

Health Canada’s Drug and Health Product Submissions Under Review (SUR) Lists are assessed using a modified approach to the selection criteria to establish a list of medicines that may have the potential to impact Canadian drug spending or clinical practice.

Medicines listed in the SUR include new drug submissions containing medicinal ingredients that have not been approved in Canada for any indication, in any strength or form. Unlike the selection of medicines identified in the pipeline lists, these medicines may have previously received market authorization through the US FDA or the EMA.

Selection Criteria

Following this initial screening, the medicine must demonstrate at least one of three selection criteria to qualify for inclusion in the report. These criteria are listed in Table 3.

Table 3. Selection criteria for the list of medicines currently under review by Health Canada

Selection Criteria
Improved safety and efficacy shown in clinical trials: a medicine that demonstrates increased safety, new outcome measures, or increased life expectancy or quality of life
Novel mechanism or First-in-class: a medicine that uses a new mechanism of biochemical interaction to produce a medical effect, or a medicine that is the first in its therapeutic class
Gene or cell therapy: a technique for the treatment of genetic disease in which a gene that is absent or defective is replaced by a healthy gene; or the transplantation of human cells to replace or repair damaged tissue and/or cells

Additional descriptive information

The profile of each medicine under review includes the key attributes listed in Table 2, as well as the indication and mechanism of action, and a summary of the applicable published outcomes from clinical trials. Specific attributes that may influence the potential uptake or cost of each medicine are also identified, as well as potential cost implications, if available, which includes the forecasted global revenues reported by GlobalData.

Although FDA designations for expedited development or review are not a selection criteria for this list, relevant Breakthrough, Fast Track, and Priority Review designations are indicated where available. For a description of these designations, see Table 1.

Indications and therapeutic areas correspond to the information provided by GlobalData. The scientific description and key attributes provided are focused on the specified indication(s) for the selected medicine. For medicines under review for multiple indications, the primary indication is used.

Emerging COVID-19 Therapies

Vaccines and medicines under development globally with an indication for COVID-19 were extracted for this section of the report, based on a development stage of Phase I, II, and III clinical trials or pre-registration. All such medicines were assessed for this analysis, both new and existing. New medicines were identified as those that have not yet been marketed for any indication, while existing medicines include previously marketed therapies undergoing evaluation for new indications related to the prevention or treatment of COVID-19.

This section also highlights the COVID-19 medicines that have been approved as well as the medicines that are currently undergoing an expedited review process with Health Canada.

Data Sources

The GlobalData Healthcare database is the primary data source for the identification of pipeline medicines and their corresponding clinical information. GlobalData Healthcare tracks medicines from pre-clinical discovery, through clinical trials, to market launch and subsequent sales. The database is a comprehensive resource of medicines under various stages of clinical development. Search capabilities allow for controlled selection of specific attributes, including but not limited to the following: phase of clinical development, therapeutic area, molecule type, indication, drug geography, mechanism of action, and regulatory designations.

Health Canada’s Drug and Health Product Submissions Under Review (SUR) Lists are used to determine the featured selection of new medicines currently undergoing review by Health Canada. The SUR is a publicly available set of lists that identify pharmaceutical and biologic drug submissions containing new medicinal ingredients not previously approved in Canada that have been accepted for review. This applies to submissions accepted on or after April 1, 2015.

As this selection is restricted to new medicines, additional sources of information are cross-referenced to confirm that the candidates have not previously been approved or sold. These include recorded sales data from the IQVIA MIDAS® Database (all rights reserved); regulatory approval records from the National Institutes of Health (NIH), US FDA, the EMA, and Health Canada; and information in Health Canada’s Clinical Trials database and ClinicalTrials.org.

Limitations

This analysis captures a snapshot of the pipeline over a specific time period. Although it is assumed to be representative of the composition of medicines over the entire year, the pipeline is fairly dynamic and the share of medicines in any particular therapeutic area will vary.

This assessment is restricted to medicines under development for market in Canada and other countries with similar regulatory and approval processes: the US and Europe (excluding Russia and Turkey). Medicines that have not yet received market authorization in these countries were considered as potential pipeline candidates, even if they have been approved elsewhere in the world.

Some of the selected medicines may be undergoing clinical trials for additional indications; this analysis only reports on indications in the late stages of development—that is, in Phase III clinical trials or pre-registration with the US FDA—that satisfy the selection criteria set out in the methodology.

For each selected pipeline medicine, the primary manufacturer(s) and trade name, if available, are given along with the indication. In some cases, additional manufacturers, including subsidiaries, may also be involved in the development of the medicine with the primary companies, or other manufacturers may be developing the same medicine for other indications.

Although this report attempts to identify the most important pipeline medicines, the selection is not exhaustive and some medicines that are not included in this selection may have a significant impact on future clinical practice and drug spending in Canada.

Unless otherwise specified, the featured lists capture the composition of the pipeline as of September 2022 and are validated as of the end of March 2023. Due to the unpredictability and fast-moving nature of pipeline medicines entering the market, some of the medicines listed in this edition may have been approved or marketed in Canada, the US, or Europe following this date. Pipeline medicines that have not been included in this report due to the timing of the selection may presently meet the selection criteria; these, along with the rest of the drug pipeline, will be considered for the next edition of the report.

Snapshot of the 2022 Pipeline

The number of new pharmaceutical developments in the pipeline is increasing year over year. In 2022, over 9,000 new medicines were undergoing clinical evaluation, which has been increasing by an average of 11% per year since 2018.

Figure 2 provides a snapshot of the pipeline including the number of new medicinal ingredients in each phase of clinical development over the last 5 years.

Figure 2. Number of new medicines in each phase of clinical evaluation, 2018-2022 Figure 2. Number of pipeline medicines in each stage of clinical evaluation
Figure description

This bar graph illustrates a snapshot of the total number of new medicines in each phase of the pipeline by their highest phase of development from 2018 to 2022. Totals are given for each year and phase.

  Phase I Phase II Phase III Pre-registration

2018

2,275

2,812

659

74

2019

2,240

2,647

629

68

2020

2,885

3,126

778

157

2021

3,582

3,733

991

154

2022

4,114

3,927

1,092

165

Data source: GlobalData Healthcare database (accessed September 2022); IQVIA MIDAS© Database.

Figure 3a illustrates the distribution of new medicines by therapeutic area from Phase I through to pre-registration. Although the findings show that pipeline medicines represented a wide range of therapeutic areas in 2022, cancer treatments dominated the therapeutic mix in each phase of the pipeline, accounting for nearly one third (30%) of medicines in all phases of clinical evaluation. Other important pipeline therapies include those for infectious diseases (such as COVID-19) and central nervous system therapies.

Figure 3b illustrates the top indications and number of medicines undergoing Phase II, Phase III or pre-registration in the major therapeutic areas in the pipeline in 2022

Figure 3a. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2022 FIGURE 3. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2020
Figure description

A stacked bar graph gives the distribution of new medicines by therapeutic rea from Phase I through to pre-registration.

Therapeutic Area Phase I Phase II Phase III Pre-registration All Phases

Oncology

39%

36%

21%

25%

30%

Infectious disease

12%

13%

21%

15%

15%

Central Nervous System

10%

9%

10%

14%

11%

Metabolic disorders

5%

5%

6%

10%

6%

Immunology

5%

5%

5%

1%

4%

Gastrointestinal

5%

4%

4%

5%

4%

Cardiovascular

3%

4%

5%

4%

4%

Respiratory

3%

3%

3%

2%

3%

Musculoskeletal disorders

2%

2%

3%

2%

2%

Dermatology

2%

3%

3%

4%

3%

Ophthalmology

2%

3%

5%

6%

4%

Genetic disorders

1%

3%

4%

3%

3%

Hematological disorders

1%

2%

4%

4%

3%

Other

9%

8%

6%

5%

8%

Data source: GlobalData Healthcare database (accessed September 2022).

Figure 3b. Top indications for major therapeutic areas in the pipeline, 2022 FIGURE 3. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2020
Figure description

A horizontal bar graph indicates the top indications by number of medicines in all phases of the pipeline.

Therapeutic Area All Phases

Oncology

30%

Non-Small Cell Lung Cancer (131)

Solid Tumor (64)

Breast Cancer (63)

Metastatic Colorectal Cancer (45)

Hepatocellular Carcinoma (43)

Acute Myelocytic Leukemia (40)

Glioblastoma Multiforme (38)

Diffuse Large B-Cell Lymphoma (32)

Infectious disease

15%

COVID-19 (172)

Human Immunodeficiency Virus (HIV) (28)

Respiratory Syncytial Virus (RSV) (15)

Hepatitis C (14)

Influenza Virus Infections (13)

Streptococcal Pneumonia (11)

Hepatitis B (9)

Central Nervous System

11%

Alzheimer’s Disease (42)

Amyotrophic Lateral Sclerosis (30)

Parkinson’s Disease (26)

Osteoarthritis Pain (16)

Migraine (15)

Major Depressive Disorder (14)

Schizophrenia (13)

Metabolic disorders

6%

Type 2 Diabetes (68)

Type 1 Diabetes (18)

Dyslipidemia (16)

Hyperlipidemia (12)

Obesity (12)

Immunology

4%

Rheumatoid Arthritis (54)

Plaque Psoriasis (38)

Graft Versus Host Disease (26)

Systemic Lupus Erythematosus (22)

Inflammation (14)

Gastrointestinal

4%

Ulcerative Colitis (35)

Crohn’s Disease (28)

NASH (17)

Liver Fibrosis (9)

Cardiovascular

4%

Hypertension (45)

Myocardial Infarction (17)

Acute Ischemic Stoke (16)

Atherosclerosis (9)

Respiratory

3%

COPD (21)

Idiopathic Fibrosis (19)

Asthma (16)

Orphan medicines, as designated by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA), accounted for a notable proportion of medicines in the 2022 pipeline. Figure 4 provides the shares of orphan designated medicines for all phases in the pipeline from 2020-2022. Orphan designated medicines make up a greater share in the later stages of the pipeline, increasing from 7% in Phase I to 31% in pre-registration in 2022. This has been a consistent trend since 2020.

Figure 4. Share of orphan medicines in the pipeline by highest phase of clinical evaluation, 2020-2022 FIGURE 3. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2020
Figure description

A stacked bar graph gives the proportion of orphan designated medicines to non-orphan designated medicines in the pipeline by phase of development from 2020 to 2022.

  Phase of Development 2020 2021 2022

Orphan designation

Phase I

8%

7%

7%

Phase II

20%

21%

22%

Phase III

33%

36%

31%

Pre-registration

40%

30%

31%

No Orphan designation

Phase I

92%

93%

93%

Phase II

80%

79%

78%

Phase III

67%

64%

69%

Pre-registration

60%

70%

69%

Note: Includes all pipeline medicines with a highest development stage of Phase I to pre-registration that are being developed for market in Canada, the United States, or geographic Europe (excluding Russia and Turkey). Orphan medicines were defined as pipeline medicines that have been granted an orphan designation by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
Data source: GlobalData Healthcare database (accessed September 2022).

Trend Watch 2022

The drug development landscape has been evolving over the last few years, from an earlier emphasis on blockbuster drugs developed in-house by large pharmaceutical companies, to niche markets and “personalized medicine”Footnote viii including various orphan drugs for rare disease, expanding therapeutic areas in gene and cell therapies, and the growing biosimilars pipeline.

Orphan drug approvals

Approvals for specialty medicines continue to increase, with orphan designated medicines accounting for 58% (29) of approved medicines in 2020 in Canada.Footnote ix This upward trend is ongoing: nearly half (49%) of the novel specialty medicines in the pipeline are indicated for orphan conditions. When including orphan cancer drugs, an estimated 80% of specialty drug development in 2022 was for orphan conditions.Footnote xi Aside from cancer drugs, another leading class of pipeline specialty drugs are novel anti-inflammatory therapeutics, including tumor necrosis factor inhibitors and targeted synthetic disease-modifying antirheumatic drugs (DMARDs).Footnote xi

Gene and cell therapies

The gene and cell therapy pipeline is growing rapidly, with more than 600 gene and cell therapies in various phases of development in 2022.Footnote x According to market analysis forecasts from GlobalData, gene therapy will be a $25 billion per year market by 2034.Footnote xi Among the many therapies undergoing clinical evaluation are novel therapies for Duchenne muscular dystrophy (6 drugs in Phase III and one in pre-registration), epidermolysis bullosa (3 drugs in preclinical evaluation, one in Phase II and one in pre-registration), and β thalassemia/sickle cell disease (10 drugs in preclinical evaluation and discovery).Footnote xii

Biosimilars pipeline

Biosimilars can provide patients and doctors with more affordable treatment options, which have the potential to provide savings and contribute to drug coverage sustainability. As of March 2023, 51 biosimilars of 16 innovator reference products have been approved in Canada. By comparison, there have been 40 approvals in the United States and 69 biosimilars approved by the European Medicines Agency.Footnote xiii The global pipeline is growing with over 140 biosimilars in Phase III and pre-registration, including new therapeutic areas such as growth hormone, infertility, bone health, and immunosuppressants. As of March 2023, Health Canada is reviewing 8 biosimilars, two of which (eculizumab and ustekinumab) are the first biosimilar submissions for the reference product.

Meds Pipeline Monitor 2022

The following tables include: selected new medicine candidates for 2022 (Table 4), retained medicines from previous editions of the Meds Pipeline Monitor (Table 5), and medicines from previous editions that have gained market authorization (Table 6).

Medicines in Phase III clinical trials or pre-registration are considered as candidates for the Meds Pipeline Monitor (MPM) if they have the potential to impact future clinical practice and drug spending in Canada (e.g., address an unmet therapeutic need, offer a novel mechanism of action or therapeutic benefit over existing therapies, or treat a serious condition).

Screening new medicine candidates

Of the 1,257 pipeline medicines in Phase III and pre-registration in 2022, twenty-eight (28) new medicines were selected for inclusion in the new medicines list (Table 4). Many of the pipeline candidates are first-in-class or represent novel mechanisms for the treatment of specific therapeutic areas. Having insight into other drugs under clinical evaluation (i.e., in Phase II) may provide additional information on the potential place in therapy of these pipeline candidates. The medicines in Phase II were examined to identify other drugs in the pipeline that have the same indication or mechanism of action as those listed in the 2022 new medicines list. The description for each new medicine listed in the 2022 new medicines list includes a statement indicating if there are any other drugs in Phase II development with the same indication or mechanism of action. Appendix A (Table A3) provides some further insights into the other drugs identified in Phase II for the indications targeted by the pipeline candidates. It is important to keep in mind that not all drugs in Phase II development will progress to Phase III. According to an industry analysis, Phase II clinical programs experience the lowest success rate of the development phases, with only 30.7% of developmental candidates advancing to Phase III. 1

Of the new medicines featured in previous reports, 25 were retained as recent evidence continues to support promising clinical benefit and satisfies the selection criteria (Table 5). Six of the 2021 pipeline medicines have received market authorization in the US, Europe, or Canada as of March 30, 2023 (Table 6), while 11 were removed from the list as their clinical trials were discontinued or they no longer fulfill the selection criteria.

Screening biosimilars

Biosimilars differ from pipeline candidates in that their efficacy and safety is similar to originator biologics. However, their introduction can substantially impact drug spending in specific therapeutic areas. Of the 21 biosimilars identified in Phase III trials in 2022, 14 (67%) would be, if approved, the first biosimilars marketed for the originator biologic. Examples include: cetuximab (for specific cancers), denosumab (for post-menopausal osteoporosis), eculizumab (for paroxysmal nocturnal hemoglobinuria), ocrelizumab (for relapsing remitting multiple sclerosis), omalizumab (for urticaria), romiplostim (for idiopathic thrombocytopenic purpura), tenecteplase (for myocardial infarction), and ustekinumab (for plaque psoriasis).

The availability of these biosimilars could significantly impact costs in a wide range of therapeutic areas. Of note, as of March 30, 2023, a biosimilar for eculizumab and ustekinumab are under review by Health Canada.2 Appendix A (Table A1) provides a list of the identified biosimilars in Phase III clinical trials and indicates whether a biosimilar currently exists for the originator biologic.

Table 4. Selected new medicines for 2022

Cardiovascular

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Abelacimab

Anthos Therapeutics Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

 

Deep vein thrombosis (DVT); Pulmonary embolism; Atrial fibrillation

 

  • Clinical trials in Canada
  • Biologic medicine
  • A dual-acting, once-monthly, fully human monoclonal antibody targeting both Factor XI and Factor XIa with high affinity and selectivity.3
  • Administered through subcutaneous and intravenous routes.
  • A single intravenous dose after total knee arthroplasty was effective for the prevention of venous thromboembolism and was associated with a low risk of bleeding.4

Clinical trials

  • Clinical trial data have shown that factor XI inhibitors like abelacimab are as effective as enoxaparin and apixaban in preventing DVT, but with a significantly lower incidence of bleeding. They can be administered daily or monthly; therefore, the monitoring interval can be longer. 5, 6
  • Two Phase III trials in cancer-associated thrombosis are ongoing.7, 8
  • There are other drugs in Phase II development (n=1) but none with the same mechanism of action as abelacimab.9 See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Aprocitentan

Idorsia Pharmaceutical Ltd

  • Increased safety and efficacy
  • Novel mechanism

Resistant hypertension

 

  • Clinical trials in Canada
  • Potential evergreening
  • A dual endothelin-receptor antagonist (ERA). It inhibits the binding of endothelin-1 to ETA and ETB receptors. By blocking the ERA receptor activation, it leads to inhibition of activity of endothelin-1 and causes vasodilation.
  • Administered orally. 

Clinical trials

  • It has demonstrated a “more favourable tolerability and safety profile in early clinical trials compared with other endothelin-receptor antagonists studied.”10 [Note: The endothelin pathway has been implicated in the pathogenesis of hypertension, but it is currently not targeted therapeutically.11]
  • Aprocitentan has a low potential for drug-drug interaction12 and may expand the therapeutic options for resistant hypertension.13
  • An NDA has been submitted to US FDA.14
  • One Phase III trial was withdrawn,15 and one has been completed.16
  • There are other drugs in Phase II development (n=3) but none with the same mechanism of action as Aprocitentan.17 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $219 million by 2028.*

Etripamil

Milestone Pharmaceuticals Inc Canada

  • Novel mechanism

Paroxysmal supraventricular tachycardia (PSVT)

 

  • Clinical trials in Canada
  • Novel administration intranasally as a nasal spray.
  • A short-acting calcium channel blocker.

Clinical trials

  • Phase II trials in moderate to high doses demonstrated efficacy comparable to the standard of care (administered at a medical facility), and took an average of 3 minutes from drug administration to conversion to sinus rhythm.18
  • It addresses an unmet medical need since there are currently no products available for out-of-hospital treatment for patients with PSVT. The only currently available acute pharmacological therapy is IV treatment with adenosine or calcium channel blockers administered in a hospital or medically supervised environment. A self-administered product for PSVT would give patients the option to safely terminate acute episodes of PSVT without the need for a hospital visit and potential admission.19
  • An analysis pooling data from the RAPID trial and the NODE-301 study indicated that the nasal spray significantly reduced medical interventions and emergency department visits.20
  • It has the potential to improve quality of life, reduce health-care burden, and alter the current management paradigm for many patients with SVT.21
  • One Phase III trial has been completed22 and others are ongoing,23, 24, 25 including an open extension study by invitation.26
  • No other drug for this indication was identified in Phase II development at this time. 27

 Forecasted revenue

  • Total global annual revenue forecasted to be $470 million by 2028.*

 

Obicetrapib

NewAmsterdam Pharma BV

  • Novel mechanism

Dyslipidemia; Heterozygous familial hypercholesterolemia (heFH);

Atherosclerosis

 

  • Clinical trials in Canada
  • Add-on therapy
  • A cholesteryl ester transfer protein (CETP) inhibitor. It has HDL-raising and LDL-C lowering effects.
  • Administered orally as a once-daily formulation.

Clinical trials

  • It has achieved significant reductions of LDL-C, up to 45%. It could become the first CETP inhibitor as add-on therapy for patients not reaching their guideline LDL-C targets.28
  • It has the potential to be a simple, once-daily, low-dose treatment option for those who are currently struggling to achieve their lipid-lowering goals on traditional therapies.29 The data to date provide evidence that it is differentiated from prior CETP inhibitors, with the potential to overcome the safety and efficacy challenges that have historically limited the potential of the drug class. 30
  • Several Phase III trials are ongoing.31, 32, 33
  • Other CETP inhibitors (dalcetrapib, evacetrapib, and anacetrapib) have been studied for this condition but none are marketed in Canada, nor are they under review by Health Canada.
  • There are other drugs in Phase II development (n=9) but none with the same mechanism of action as obicetrapib. 34 See Appendix A for additional information.

 Forecasted revenue

  • Total global annual revenue forecasted to be $222 million by 2028.*

Sotatercept

Acceleron Pharma Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Pulmonary arterial hypertension (PAH)

  • Clinical trials in Canada
  • Add-on therapy
  • Rare or orphan designation
  • Biologic medicine
  • An activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein that increases hemoglobin levels and red blood cells.35, 36
  • Administered subcutaneously and intravenously.

Clinical trials

  • In the Phase 3 STELLAR study, when added to background therapy, it showed a clinically significant effect on the primary efficacy outcome measure of improvement from baseline to 24 weeks in six-minute walk distance.37
  • Despite some hurdles (such as low uptake since the initial five doses, administered subcutaneously every 21 days, require specialty infrastructure lacking in community centres), knowledge opinion experts say it could become a leading therapy for PAH due to strong efficacy and clinical data, and as the first disease-modifying therapy, a significant breakthrough for PAH patients.38
  • Several Phase III trials are ongoing.39, 40, 41, 42
  • There are other drugs in Phase II development (n=8) but none with the same mechanism of action as sotatercept. 43 See Appendix A for additional information.

 Forecasted revenue

  • Total global annual revenue forecasted to be $1.8 billion by 2028.*

 

Central Nervous System

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Evobrutinib

Merck KGaA

  • Increased safety and efficacy
  • Novel mechanism

 

Relapsing multiple sclerosis (RMS); Secondary progressive multiple sclerosis (SPMS)

  • Clinical trials in Canada
  • A selective, central nervous system (CNS) penetrant immunomodulator that irreversibly blocks Bruton’s tyrosine kinase (BTK) which suppresses the autoimmunity associated with the disease.
  • Administered orally.

Clinical trials

  • No bleeding has been reported in clinical trials to date (in multiple sclerosis (MS)).44
  • There is evidence that it crosses the blood-brain barrier and may be superior to currently available disease-modifying therapies at dampening the chronic neuroinflammatory processes compartmentalized within the CNS that contribute to progressive worsening in people with MS.45
  • It has the potential to become a safe and highly efficacious treatment option for RMS by addressing both peripheral and central drivers of inflammation through inhibition of BTK. Its dual-faceted approach may offer better control of silent progression of the disease in between attacks on top of strong relapse control in people living with RMS.46
  • Two Phase III trials were terminated47, 48 and two others are ongoing.49, 50
  • There are other drugs in Phase II development (n=9) but none with the same mechanism of action as evobrutinib. 51See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $798 million by 2028.*

Reldesemtiv

Cytokinetics Inc

  • Increased safety and efficacy
  • Novel mechanism

Amyotrophic lateral sclerosis (ALS)

  • Clinical trials in Canada
  • Rare or orphan designation
  • A fast skeletal muscle troponin activator that slows the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibres, leading to an increase in skeletal muscle contractility.52, 53
  • Administered orally.

Clinical trials

  • Although the primary efficacy analysis in a Phase II study did not demonstrate statistical significance, there were trends favouring reldesemtiv, with effect sizes generally regarded as clinically important.54, 55
  • Additional analyses suggested that those with shorter disease duration and moderate-to-fast progression at study start may experience the greatest benefits.56, 57
  • Results suggest ALS patients receiving reldesemtiv may have a lower risk of and delayed need for durable medical equipment related to impaired mobility, breathing, swallowing, or speaking; this delay is consistent with other measures indicating delay in disease progression.58
  • Phase III trials are ongoing.59, 60
  • There are other drugs in Phase II development (n>10) but none with the same mechanism of action as reldesemtiv.61 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $215 million by 2028.*

Soticlestat

Takeda Pharmaceutical Co Ltd

  • Increased safety and efficacy
  • Novel mechanism

Lennox-Gastaut syndrome; Dravet syndrome (severe myoclonic epilepsy of infancy)

  • Clinical trials in Canada
  • Add-on therapy
  • Rare or orphan designation
  • It inhibits the activity of cholesterol 24-hydroxylase, one of several enzymes responsible for catabolism of cholesterol. Inhibition of the enzyme lowers the level of 24S-hydroxycholesterol, a brain-specific metabolite that modulates a wide variety of receptors and ion channels, including the N-methyl-D-aspartate receptor (NMDA) receptor.62
  • Administered orally.

Clinical trials

  • It can ameliorate seizure symptoms through a mechanism distinct from conventional antiseizure medications.63
  • In a Phase II trial, treatment resulted in statistically significant, and clinically meaningful reductions from baseline in median seizure frequency and in convulsive seizure frequency.64
  • Phase III trials are ongoing.65, 66, 67
  • There are other drugs in Phase II development (n=2) but none with the same mechanism of action as soticlestat. 68 See Appendix A for additional information.

 Forecasted revenue

  • Total global annual revenue forecasted to be $241 million by 2028.*

Ulotaront (SEP-363856)

Sunovion Pharmaceuticals Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Schizophrenia

  • A trace amine-associated receptor 1 (TAAR1) and serotonin 5-HT1A receptors agonist.
  • Administered orally.

Clinical trials

  • In a Phase II study, it demonstrated sustained improvements in the negative symptoms of anhedonia and avolition in patients with schizophrenia between the ages of 18 and 40. Patients who took part in the 26-week, open-label extension study, showed a reduction in the total Positive and Negative Syndrome Scale (PANSS) score.69
  • It seems to lack the weight gain, metabolic issues, and extrapyramidal symptoms associated with traditional antipsychotics.70
  • It is not likely to pose a risk for recreational abuse and may have potential therapeutic utility as a treatment of substance use disorders.71
  • Several Phase III trials are ongoing.72, 73, 74, 75, 76, 77, 78
  • There are other drugs in Phase II development (n=9) with one having the same mechanism of action as ulotaront. 79 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $507 million by 2028.*

Dermatology

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Beremagene geperpavec

Krystal Biotech Inc

  • Novel mechanism
  • Gene or cell therapy
  • Priority Review
  • Fast Track

Epidermolysis bullosa

 

  • Biologic medicine
  • Rare or orphan designation
  • A gene therapy consisting of a replication-defective, non-integrating viral vector, engineered to deliver functional human COL7A1 genes. 80
  • It is administered as a topical gel.
  • There are no approved therapies for this serious condition.

Clinical trials

  • The results of a Phase III study81 showed that its use improved wound healing with good tolerability over six months, with 67% of treated wounds completely healed at six months, as compared with 22% of the wounds given a placebo gel. 82, 83  The study was limited by a relatively small sample size (n=31) and use of the agent in small wounds. It is also lacking in longer term information.84
  • An open-label study to assess long term safety is recruiting.85
  • No other drug for this indication was identified in Phase II development at this time.
  • There are no Canadian clinical trials currently listed in Health Canada’s clinical trial database.

Forecasted revenue

  • Total global annual revenue forecasted to be $613 million by 2028.*

Gastrointestinal Disorders

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Resmetirom

Madrigal Pharmaceuticals Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough
  • Fast Track

Non-alcoholic steatohepatitis (NASH); Non-alcoholic fatty liver disease (NAFLD)

  • Clinical trials in Canada
  • It acts as a liver-directed thyroid hormone receptor (THR)-beta agonist.
  • Administered orally.
  • There are no approved therapies specifically for NASH/NAFLD.86 Many agents are being investigated.

Clinical trials

  • In a Phase II study, it was associated with greater improvements in physical functioning and Physical Component Summary (PCS) scores. Patients with improvement in NASH and fibrosis on liver biopsy also showed improvement in components of health-related quality of life (HRQL).87
  • The unpublished results of the Phase IIIb MAESTRO-NAFLD-1 trial demonstrated that it appears safe, well-tolerated and may provide statistically significant improvements in key measures of liver and cardiovascular health. 88
  • Several Phase III trials are ongoing.89, 90, 91, 92
  • There are other drugs in Phase II development (n>10) with several having the same mechanism of action as resmetirom. 93 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $1.5 billion by 2028.*

Seladelpar lysine

CymaBay Therapeutics Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Primary biliary cholangitis (primary biliary cirrhosis)

  • Clinical trials in Canada
  • Rare or orphan designation
  • A selective, peroxisomal proliferator-activated receptor delta (PPAR-delta) agonist.
  • Administered orally.

Clinical trials

  • Treatment options for primary biliary cholangitis (PBC) are limited.94
  • Seladelpar treatment for 1 year led to consistent improvement in both symptom burden and biochemical response, suggesting its potential as a single agent to address key unmet needs in PBC patients. 95, 96
  • It appeared safe and well tolerated and was not associated with any increase in pruritus. 97
  • One Phase III trial was completed98 and others are ongoing.99, 100
  • There are other drugs in Phase II development (n=6) at this time, but none with the same mechanism of action as seladelpar.101 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $704 million by 2028.*

Genetic disorders

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Delandistrogene moxeparvovec

Sarepta Therapeutics Inc

  • Novel mechanism
  • Gene or cell therapy
  • Priority Review
  • Fast Track

Duchenne muscular dystrophy

 

  • Biologic medicine
  • Rare or orphan designation
  • It constitutes micro dystrophin gene carried through recombinant adeno-associated virus vector serotype 74 (AAV74). It acts by activating dystrophin.102
  • It is administered intramuscularly and intravenously.

Clinical trials

  • In clinical results from more than 80 treated patients, it has demonstrated positive results at multiple time points, including one-, two- and up to four-years after treatment, in addition to demonstrating a consistent safety profile.103
  • A Phase III trial is ongoing.104
  • No other drug for this indication was identified in Phase II development at this time.
  • There are no Canadian clinical trials currently listed in Health Canada’s clinical trial database.

Forecasted revenue

  • Total global annual revenue forecasted to be $2.9 billion by 2028.*

REC-2282

Recursion Pharmaceuticals Inc

  • Increased safety and efficacy
  • Fast Track

Neurofibromatosis type II (NF2)-mutated meningiomas

  • Rare or orphan designation
  • A pan-histone deacetylase (HDAC) inhibitor. It inhibits both histone and non-histone proteins.
  • Administered orally.

Clinical trials

  • In studies to date, it appears to be well tolerated, including in patients dosed for multiple years. 105
  • Its oral bioavailability, CNS penetrance, and potentially reduced cardiac toxicity would differentiate it from other HDAC inhibitors (e.g., vorinostat, romidepsin).106
  • If successful in the Phase III trials, it could be the first approved treatment for NF2-mutated meningiomas.107
  • A Phase II/III trial is ongoing.108
  • There is one other drug in Phase II development but not with the same mechanism as REC-2282.109 See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Immunological disorders

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Garadacimab

CSL Ltd

  • Increased safety and efficacy
  • Novel mechanism

Hereditary angioedema (HAE) (C1 esterase inhibitor [C1-INH] deficiency

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine
  • A recombinant, fully human, immunoglobulin G4 monoclonal antibody that interferes with FXIIa-mediated coagulation. By targeting FXIIa, it inhibits the HAE cascade at its origin as compared with other HAE therapies that target downstream mediators.
  • Administered as a once-monthly subcutaneous injection and intravenously.

Clinical trials

  • In a Phase III study, it reduced the number of attacks for up to six months and demonstrated favourable safety and tolerability. 110
  • It has the potential to become a transformative first-in-class therapy for people living with HAE. 111
  • One Phase III trial was completed112 and another one is ongoing.113
  • There are other drugs in Phase II development at this time (n=3), 114 but none with the same mechanism as garadacimab. See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Omidubicel

Gamida Cell Ltd

  • Novel mechanism
  • Breakthrough
  • Gene or cell therapy
  • Priority Review

 

Hematopoietic stem cell transplantation

  • Rare or orphan designation
  • Biologic medicine
  • It is an ex vivo expanded hematopoietic progenitor cell and nonexpanded myeloid and lymphoid cell product derived from a single umbilical cord blood unit.115
  • It is administered as an infusion.

Clinical trials

  • Transplantation with omidubicel results in faster hematopoietic recovery and reduces early transplant-related complications compared with standard umbilical cord blood transplantation (UCBT). 116, 117
  • One-year post-transplant data showed sustained clinical benefits as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease rates.118
  • It has the potential to become the standard of care for adult patients eligible for UCBT. 119
  • No other drug for this indication was identified in Phase II development at this time.
  • There are no Canadian clinical trials currently listed in Health Canada’s clinical trial database.

Forecasted revenue

  • Total global annual revenue forecasted to be $121 million by 2028.*

Infectious Diseases

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Posoleucel

AlloVir Inc

  • Novel mechanism
  • Gene or cell therapy

Herpesviridae infections

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine
  • An allogeneic, off-the-shelf multi-virus specific T-cell therapy.
  • It is administered by intravenous injection and by infusion.

Clinical trials

  • Currently, there are no approved therapies for most viral infections in the post-stem cell transplant setting, with the standard of care treatments having limited efficacy and associated with significant toxicity.120
  • Phase II data showed a substantial reduction in the expected rate of clinically significant viral infections or diseases in the high-risk patient population despite the expected high rates of viral reactivation.121
  • No other drug for this indication was identified in Phase II development at this time.
  • Phase III trials are ongoing. 122, 123, 124

Forecasted revenue

  • Total global annual revenue forecasted to be $1.4 billion by 2028.*

Zoliflodacin

Entasis Therapeutics Holdings Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

Uncomplicated cervical and urethral gonorrhea

  • The first in a new class: the spiropyrimidinetriones. It is a DNA gyrase and topoisomerase IV inhibitor that leads to disruption of DNA synthesis and subsequently cell death.
  • Administered orally, as a single dose.

Clinical trials

  • Its cardiac safety was evaluated and there were no clinically significant effects on heart rate, PR and QRS intervals, electrocardiogram morphology, or laboratory values, confirming that a single dose is safe and well tolerated.125
  • It has a unique mode of inhibition against bacterial type II topoisomerases with binding sites in bacterial gyrase that are distinct from those of the fluoroquinolones. It represents “a promising new oral therapy for drug-resistant infections caused by N. gonorrheae”. 126
  • A Phase III trial is ongoing.127
  • No other drug for this indication was identified in Phase II development at this time.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Metabolic Disorders

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Insulin human, oral

(ORMD-0801)

Oramed Pharmaceuticals Inc

  • Novel mechanism

 

Type 2 Diabetes

 

  • Clinical trials in Canada
  • Biologic medicine
  • An insulin receptor agonist.
  • Administered orally.

Clinical trials

  • It has the potential to significantly impact therapy of type 2 diabetes.
  • Phase III trials are ongoing.128, 129, 130
  • There is an inhalational insulin human but are no other oral Insulins identified in Phase II development at this time.

Forecasted revenue

  • Total global annual revenue forecasted to be $394 million by 2028.*

Insulin icodec

Novo Nordisk AS

  • Novel mechanism

Type 1 diabetes (juvenile diabetes); Type 2 diabetes

 

  • Clinical trials in Canada
  • Potential evergreening
  • Biologic medicine
  • A long-acting basal insulin analogue.
  • Administered subcutaneously once weekly.

Clinical trials

  • In a Phase II clinical trial, once-weekly insulin icodec provided safe and efficacious glycemic control comparable to once-daily insulin glargine in type 2 diabetes patients.131, 132
  • A reduction in the frequency of basal insulin injections might facilitate treatment acceptance and adherence among patients with type 2 diabetes.133
  • Several Phase III trials have been completed134, 135, 136, 137 and many are ongoing.138, 139, 140, 141, 142
  • There is no other once-weekly insulin identified in Phase II development at this time.

Forecasted revenue

  • Total global annual revenue forecasted to be $973 million by 2028.*

Oncology

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Bemarituzumab

Amgen Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Adenocarcinoma of the gastroesophageal junction; Gastric cancer; Bladder cancer; Gastroesophageal (GE) junction carcinomas

  • Clinical trials in Canada
  • Biologic medicine
  • A targeted monoclonal antibody that is designed to block fibroblast growth factors (FGFs) from binding and activating fibroblast growth factor receptor 2b (FGFR2b), inhibiting several downstream pro-tumour signaling pathways and potentially slowing cancer progression.
  • Administered intravenously.

Clinical trials

  • In the Phase II FIGHT trial, adding it to chemotherapy led to longer progression-free survival and overall survival in patients with advanced FGFR2b-positive gastric or gastroesophageal junction cancers. It was also associated with more side effects, including eye problems.143
  • Phase III trials are ongoing.144, 145
  • There are other drugs in Phase II development at this time (n>10),146 including one drug with a similar mechanism of action to bemarituzumab. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $639 million by 2028.*

Iberdomide hydrochloride

Bristol-Myers Squibb Co

  • Increased safety and efficacy
  • Novel mechanism

Refractory multiple myeloma; Relapsed multiple myeloma

  • Clinical trials in Canada
  • Rare or orphan designation
  • An immunomodulatory imide drug that targets cereblon (CRBN) and inhibits CRBN ubiquitination and enhances T cell IL-2 production, but inhibits B-cell production of immunoglobulin.
  • Administered orally.

Clinical trials

  • Iberdomide plus dexamethasone was generally safe and showed meaningful clinical activity in heavily pretreated patients with multiple myeloma, including in disease that was refractory to immunomodulatory drugs.147
  • Phase III trials are ongoing.148, 149
  • There are other drugs in Phase II development at this time (n>10),150 including two drugs with a similar mechanism of action to iberdomide. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $276 million by 2028.*

Imetelstat sodium

Geron Corp

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

Myelodysplastic syndrome; Post-essential thrombocythemia myelofibrosis (post-ET MF); Post-polycythemia vera myelofibrosis (PPV-MF)

  • Clinical trials in Canada
  • Rare or orphan designation
  • A lipid-conjugated oligonucleotide that binds directly with high affinity to the template region of the RNA component of human telomerase (hTR) and inhibits telomerase enzymatic activity.
  • Administered by intravenous infusion.

Clinical trials

  • It demonstrated meaningful clinical benefit including a robust symptom response rate and potential overall survival benefit in IMbark, a Phase II study in intermediate-2 or high-risk myelofibrosis patients who have relapsed after or are refractory to Janus kinas inhibitors.151, 152
  • Phase III trials are ongoing.153, 154  
  • There are other drugs in Phase II development at this time (n>10),155 but none with the same mechanism as imetelstat. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $945 million by 2028.*

Navitoclax dihydrochloride

AbbVie Inc

  • Increased safety and efficacy
  • Novel mechanism

Myelofibrosis

  • Clinical trials in Canada
  • Rare or orphan designation
  • It acts as an antagonist of a subset of the B-cell leukemia 2 (Bcl-2) family of proteins. It selectively binds to apoptosis suppressor proteins Bcl-2, Bcl-w and Bcl-XL and prevents their binding to the apoptotic effectors Bax and Bak proteins, which may trigger apoptosis in tumour cells overexpressing Bcl-2 and Bcl-XL.
  • Administered orally.

Clinical trials

  • The clinical data to date, especially in synergistic combination with traditional Janus kinas 2 (JAK2) inhibitors, have been promising for those with refractory or relapsing disease on prior therapies.156
  • Its addition to ruxolitinib in patients with persistent or progressive myelofibrosis resulted in durable spleen volume reduction, improved total symptom score, hemoglobin response, and bone marrow fibrosis.157
  • Phase III trials are ongoing.158, 159
  • There are other drugs in Phase II development at this time (n>10),160 but none with the same mechanism as navitoclax. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $611 million by 2028.*

Relacorilant

Corcept Therapeutics Inc

  • Increased safety and efficacy
  • Novel mechanism

Epithelial ovarian cancer

  • Clinical trials in Canada
  • A selective glucocorticoid receptor antagonist that targets glucocorticoid receptor II (GR-II).
  • Administered orally.

Clinical trials

  • Results of a Phase II study showed improvements in progression free survival, duration of response and overall survival without increased side effect burden.161
  • As there are no currently approved therapies or effective standard of care for heavily pretreated patients with ovarian cancer who have exhausted single-agent chemotherapy and/or bevacizumab, the combination of intermittently administered relacorilant and nab-paclitaxel may demonstrate a substantial improvement without increased toxicity compared with nab-paclitaxel.162
  • If the Phase III study confirms these results, “relacorilant plus nab-paclitaxel has the potential to become a new standard of care.”163
  • The Phase III trial in ovarian cancer is ongoing.164
  • There are other drugs in Phase II development at this time (n>10),165 but none with the same mechanism as relacorilant. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $455 million by 2028.*

Rusfertide acetate

Protagonist Therapeutics Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough
  • Fast Track

 

 

Polycythemia vera (PV)

 

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine
  • A synthetic mimetic of hepcidin, a natural hormone that regulates iron absorption, distribution, and storage, controlling the body’s production of red blood cells.
  • Administered weekly as a subcutaneous injection.
  • It has been suggested to offer ‘greater potency, stability, and solubility than with the natural hormone.’166 

Clinical trials

  • In a Phase II study, its administration provided PV patients with an effective therapy that led to rapid and sustained hematocrit control, and potentially a better quality of life by keeping them essentially phlebotomy-free for up to 18 months.167, 168
  • It has the potential to provide a highly effective treatment option for patients with PV, providing an opportunity to fundamentally transform the management of this disease.169, 170
  • A Phase III trial is ongoing.171
  • There are no other drugs for this indication in Phase II development at this time.172

Forecasted revenue

  • Forecasted annual global revenue unknown.

Zolbetuximab

Astellas Pharma Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

 

Adenocarcinoma of the gastroesophageal junction; Gastric cancer

 

  • Clinical trials in Canada
  • Add-on therapy
  • Biologic medicine
  • A monoclonal antibody that targets and binds to claudin 18.2 (CLDN18.2, a transmembrane protein).
  • Administered as an intravenous infusion.  

Clinical trials

  • It yielded positive results with few high-grade adverse events; thus, it has the potential to be a novel and effective therapy.173 
  • Claudin 18.2 (CLDN 18.2) is overexpressed in at least a third of esophagogastric adenocarcinomas. The combination of zolbetuximab and chemotherapy provides a survival benefit, correlated with the intensity of CLDN 18.2 expression.174
  • Results from one Phase III trial showed that both progression-free and overall survival were significantly improved with zolbetuximab plus mFOLFOX6 treatment.175
  • Phase III trials are ongoing.176, 177
  • There are other drugs in Phase II development at this time (n>10), 178 including two drugs with a similar mechanism of action to zolbetuximab. See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $622 million by 2028.*

Ophthalmology

Medicine (Trade name) Company Indication(s) Description and Key Attributes

Lenadogene nolparvovec

GenSight Biologics SA

  • Novel mechanism
  • Gene or cell therapy

 

Leber’s hereditary optic neuropathy (Leber optic atrophy)

 

  • Biologic medicine
  • Rare or orphan designation
  • A recombinant adeno-associated virus vector serotype 2 (AAV2) containing the human wild-type mitochondrial ND4 gene (rAAV2-ND4 vector).
  • It is administered intravitreally.

Clinical trials

  • Leber hereditary optic neuropathy (LHON) remains a disease with a high unmet medical need.179
  • Its efficacy in improving visual acuity in LHON was confirmed in a large cohort of unilaterally-treated patients,180, 181, 182 compared to the spontaneous natural history decline.183, 184
  • It has a good overall safety profile with excellent systemic tolerability, consistent with limited bio-dissemination.185
  • There is an unresolved question about whether bilateral injection could offer added benefits over unilateral injection. The FDA has requested that the company conduct an additional trial.186
  • There are no other drugs for this indication in Phase II development at this time.
  • There are no Canadian clinical trials currently listed in Health Canada’s clinical trial database.

Forecasted revenue

  • Forecasted annual global revenue unknown.

* Consensus forecasts for global revenue data were collected from GlobalData, Q4-2022, and are given in US dollars.
Data source: GlobalData Healthcare database.

Table 5. Update on pipeline medicines retained from the 2021 Meds Pipeline Monitor

Cardiovascular

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Apabetalone

Resverlogix Corp.

  • Novel mechanism
  • Breakthrough

Coronary artery disease (CAD)

(ischemic heart disease)

  • Clinical trials in Canada

Clinical trials

  • Positive results from the Phase III have been published.187 There is no information available at this time to confirm if an application has been submitted.

Forecasted revenue

  • Forecasted annual global revenue unknown.

CSL112

CSL Ltd

  • Increased safety and efficacy
  • Novel mechanism

Acute coronary syndrome (ACS)

Clinical trials

  • Phase III trial is still ongoing; targeted to be completed in December 2023. 188
  • According to the Memorial Care Heart and Vascular Institute that is participating in the Phase III trial, “this infusion therapy is a game changer because we are able to administer this potentially lifesaving intravenous treatment directly into the bloodstream soon after the onset of a cardiac event, allowing us the potential to clear the blockages that cause heart attacks”.189

Forecasted revenue

  • Forecasted annual global revenue unknown.

Central Nervous System

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Latozinemab
(previously AL-001)

Alector Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

   

Frontotemporal dementia (FTD)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Clinical trials

  • The Phase III trial is still ongoing; targeted to be completed in December 2023. 190

Forecasted revenue

  • Total global annual revenue forecasted to be $333 million by 2028.*

Valiltramiprosate
(previously ALZ-801)

Alzheon Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

   

Alzheimer's disease (AD)

Clinical trials

  • The Phase III trial is still ongoing; targeted to be completed in July 2024. 191

Forecasted revenue

  • Forecasted annual global revenue unknown.

Midomafetamine (MDMA)

Multidisciplinary Association for Psychedelic Sudies

  • Increased safety and efficacy
  • Novel mechanism

Post-traumatic stress disorder (PTSD)

  • Clinical trials in Canada

Clinical trials

  • A review article described MDMA-assisted psychotherapy as an effective therapy for patients with PTSD with a reasonable safety profile.192
  • Another Phase III trial has been completed;193 the other is enrolling by invitation, until March 2023.194

Forecasted revenue

  • Total global annual revenue forecasted to be $75 million by 2028.*

ND-0612
(levodopa/carbidopa for subcutaneous infusion)

Mitsubishi Tanabe Pharma Corp

  • Increased safety and efficacy

Parkinson's disease (PD)

  • Add-on therapy

Clinical trials

  • The Phase III trial is still ongoing; targeted to be completed in October 2023.195

Forecasted revenue

  • Forecasted annual global revenue unknown.

Gastrointestinal disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Brazikumab

AstraZeneca Plc

  • Increased safety and efficacy

Crohn's disease

(regional enteritis)

  • Clinical trials in Canada
  • Biologic medicine

Clinical trials

  • Phase III trials are still ongoing.196, 197

Forecasted revenue

  • Total global annual revenue forecasted to be $377 million by 2028.*

RBX-2660

Rebiotix Inc

  • Increased safety and efficacy

Clostridium difficile infections (C. difficile associated disease)

  • Clinical trials in Canada

Clinical trials

  • A Phase III trial has been completed198 and the results published.199 It found that “RBX2660 is a safe and effective treatment to reduce recurrent C. difficile infection following standard-of-care antibiotics with a sustained response through 6 months. It is the first microbiota-based live biotherapeutic to demonstrate efficacy as early as first recurrence of Clostridioides difficile (C. difficile) infection.” 200
  • Another Phase III trial is still ongoing; targeted to be completed in September 2023. 201

Forecasted revenue

  • Forecasted annual global revenue unknown.

Genito urinary system and sex hormones

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Gepotidacin mesylate

GlaxoSmithKline Plc

  • Increased safety and efficacy
  • Novel mechanism

Cystitis; Urinary tract infections (UTI)

Clinical trials

  • The pivotal Phase III trials have stopped enrolment early for efficacy following a recommendation by the Independent Data Monitoring Committee.202, 203 This decision was based on a pre-specified interim analysis of efficacy and safety data in over 3000 patients across the trials. The EAGLE-2 and 3 trials are now closed for recruitment, with final study visits and data collection anticipated during the first quarter of 2023.204, 205
  • The company expects to submit regulatory filings to the US FDA in the first half of 2023.206

Forecasted revenue

  • Total global annual revenue forecasted to be $376 million by 2028.*

Hematological disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Bentracimab

PhaseBio Pharmaceuticals Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Bleeding and clotting disorders

  • Biologic medicine

Clinical trials

  • Interim Phase III results positive. 207
  • The company plans to submit a BLA to the US FDA in late 2022. 208

Forecasted revenue

  • Total global annual revenue forecasted to be $436 million by 2028.*

Danicopan

Alexion Pharmaceuticals Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Paroxysmal nocturnal hemoglobinuria (PNH)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Add-on therapy

Clinical trials

  • An interim analysis of the Phase III trial showed positive high-level results in patients with PNH who experience clinically significant extravascular haemolysis.209
  • The Phase III trial is ongoing; targeted to be completed in December 2023.210

Forecasted revenue

  • Total global annual revenue forecasted to be $109 million by 2028.*

Fidanacogene
elaparvovec

Pfizer Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Gene or cell therapy
  • Breakthrough

Hemophilia B
(factor IX deficiency)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Clinical trials

  • The US FDA accepted a BLA in October 2022; a decision is expected by June 2023.211

Forecasted revenue

  • Total global annual revenue forecasted to be $364 million by 2028.*

Fitusiran

Sanofi

  • Increased safety and efficacy
  • Fast Track

Hemophilia A;
Hemophilia B

  • Clinical trials in Canada
  • Rare or orphan designation

Clinical trials

  • Results from a Phase III trial are positive.212 Further investigation under an amended protocol with lower doses and a less frequent dosing regimen (every other month) is underway. 213
  • A Phase III long-term study, until October 2026, is ongoing.214

Forecasted revenue

  • Total global annual revenue forecasted to be $415 million by 2028.*

Hormonal disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Palopegteriparatide

(TransCon PTH)

Ascendis Pharma AS

  • Increased safety and efficacy
  • Priority Review

Hypoparathyroidism

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Clinical trials

  • Results from the Phase III trial have been published.215
  • A NDA has been submitted to the US FDA and was granted Priority review.216

Forecasted revenue

  • Total global annual revenue forecasted to be $1.1 billion by 2028.*

Infectious diseases

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

V-7

Immunitor Inc

  • Increased safety and efficacy
  • Novel mechanism

Tuberculosis (TB)

  • Rare or orphan designation
  • Add-on therapy

Clinical trials

  • No further updates.
  • It is one of many in the tuberculosis pipeline.217

Forecasted revenue

  • Forecasted annual global revenue unknown.

Male health

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Fexapotide triflutate

Nymox Pharmaceutical Corp

  • Increased safety and efficacy
  • Novel mechanism

Benign prostatic hyperplasia (BPH)

  • Biologic medicine

Clinical trials

  • An NDA has been submitted to the US FDA.218

Forecasted revenue

  • Forecasted annual global revenue unknown.

Metabolic Disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Birtamimab

Prothena Corp Plc

  • Increased safety and efficacy
  • Fast Track

Primary systemic amyloidosis

  • Rare or orphan designation
  • Add-on therapy
  • Biologic medicine

Clinical trials

  • Results from the Phase III (AFFIRM-AL) trial will be presented.219
  • The Phase III trial is ongoing; targeted to be completed in June 2024.220

Forecasted revenue

  • Total global annual revenue forecasted to be $193 million by 2028.*

Donislecel

(Lantidra)

CellTrans Inc

  • Increased safety and efficacy
  • Gene or cell therapy

Type 1 diabetes (T1D; juvenile diabetes)

  • Biologic medicine

Clinical trials

  • A BLA has been submitted to the US FDA. The FDA Advisory Committee voted 12 yes / 4 no (one abstention) that donislecel delivered by intraportal administration has an overall favorable benefit-risk profile for some patients with type 1 diabetes.221

Forecasted revenue

  • Forecasted annual global revenue unknown.

Pegunigalsidase alfa

Chiesi Farmaceutici SpA

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

Fabry disease
(FD)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Clinical trials

  • Company met with the US FDA to discuss issues from previous application222 and resubmitted their data.223 It is also being reviewed by the EMA.224

Forecasted revenue

Forecasted annual global revenue unknown.

Oncology

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Arfolitixorin

Isofol Medical AB

  • Increased safety and efficacy
  • Fast Track

Metastatic colorectal cancer

  • Clinical trials in Canada
  • Add-on therapy

Clinical trials

  • The Phase III trial is still ongoing; targeted to be completed in January 2023.225

Forecasted revenue

  • Forecasted annual global revenue unknown.

SGX-301
(synthetic hypericin)

(HyBryte)

Soligenix Inc

  • Increased safety and efficacy
  • Fast Track

Cutaneous T-cell lymphoma (CTCL)

  • Rare or orphan designation

Clinical trials

  • The Phase III trial is completed 226 and results have been published.227
  • The company has filed an NDA; the US FDA issued a refusal letter on the basis that it was insufficient to allow for substantive review.228 The company plans to meet with the US FDA.

Forecasted revenue

  • Total global annual revenue forecasted to be $8 million by 2028.*

Ipatasertib

Genentech, Inc

  • Increased safety and efficacy
  • Novel mechanism

Metastatic
hormone
refractory
(castration resistant,
androgen independent)
prostate cancer

  • Clinical trials in Canada
  • Biologic medicine
  • Add-on therapy

Clinical trials

  • A Phase III trial is still ongoing; targeted to be completed in October 2023.229

Forecasted revenue

  • Total global annual revenue forecasted to be $424 million by 2028.*

Motixafortide

(BL-8040; Aphexda)

BioLineRx Ltd

  • Increased safety and efficacy
  • Novel mechanism

Multiple myeloma (Kahler disease)

  • Clinical trials in Canada
  • Add-on therapy
  • Biologic medicine

Clinical trials

  • The Phase III (GENESIS) trial is completed. Results showed that nearly 90% of patients collected an optimal number of cells for transplantation following a single administration and in only one apheresis session. 230
  • An NDA has been submitted to the US FDA.231

Forecasted revenue

  • Total global annual revenue forecasted to be $70 million by 2028.*

Ophthalmology

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Avacincaptad pegol sodium

(Zimura)

Iveric Bio Inc

  • Increased safety and efficacy
  • Fast Track
  • Priority Review
  • Breakthrough

Geographic atrophy (GA)

  • Clinical trials in Canada

Clinical trials

  • Phase III trial is still ongoing; targeted to be completed in July 2023.232
  • The company has submitted an NDA to the US FDA and it has been accepted for review. 233

Forecasted revenue

  • Total global annual revenue forecasted to be $1.5 billion by 2028.*

Women’s health

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Fezolinetant

Astellas Pharma Inc

  • Increased safety and efficacy
  • Novel mechanism
  • Priority Review

Vasomotor symptoms of menopause (hot flashes)

  • Clinical trials in Canada

Clinical trials

  • An NDA has been submitted to the US FDA, with a target review date of February 22, 2023.234 [Note: Results from a Phase III trial in Asia did not meet statistical significance.235 This may impact the FDA review.]
  • The US FDA review, due February 22, 2023, has been extended by 3 months.236

Forecasted revenue

  • Total global annual revenue forecasted to be $1.8 billion by 2028.*

* Consensus forecasts for global revenue data were collected from GlobalData, Q4-2022, and are given in US dollars.
Data source: GlobalData Healthcare database.

Table 6. Pipeline medicines from the 2021 Meds Pipeline Monitor that have gained market authorization

Central Nervous System

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Lecanemab

(Leqembi)

Eisai Co Ltd

  • Increased safety and efficacy
  • Priority Review
  • Fast Track
  • Breakthrough

Alzheimer's disease (AD)

  • Clinical trials in Canada
  • Biologic medicine

Approval

  • Approved by the US FDA (Leqembi; January 6, 2023).237

 Forecasted revenue

  • Total global annual revenue forecasted to be $4.7 billion by 2028.*

Ublituximab

(Briumvi)

TG Therapeutics, Inc.; LFB S.A.

  • Increased safety and efficacy

Relapsing multiple sclerosis (RMS)

  • Clinical trials in Canada
  • Biologic medicine
  • Add-on therapy

Approval

  • Approved by the US FDA (Briumvi; December 28, 2022).238

 Forecasted revenue

  • Total global annual revenue forecasted to be $826 million by 2028.*

Hematological disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Etranacogene dezaparvovec

(Hemgenix)

CSL Ltd

  • Increased safety and efficacy
  • Novel mechanism
  • Gene or cell therapy
  • Breakthrough
  • Priority Review

Hemophilia B (factor IX deficiency)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Approval

  • Approved by the US FDA (Hemgenix; November 22, 2022).239

 Forecasted revenue

  • Total global annual revenue forecasted to be $386 million by 2028.*

Infectious Disease

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Oteseconazole

(Vivjoa)

Mycovia Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Fast Track
  • Priority Review

Recurrent vulvovaginal candidiasis (RVVC)

  • Clinical trials in Canada

Approval

  • Approved by the US FDA (Vivjoa; April 26, 2022) 240

 Forecasted revenue

  • Forecasted annual global revenue unknown.

Metabolic Disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Teplizumab

(Tzield)

Provention Bio Inc and Sanofi

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

 

Type 1 diabetes
(T1D; juvenile diabetes)

  • Clinical trials in Canada
  • Rare or orphan designation
  • Biologic medicine

Approval

  • Approved by the US FDA (Tzield; November 17, 2022).241

 Forecasted revenue

  • Total global annual revenue forecasted to be $604 million by 2028.*

Oncology

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Elacestrant

(Orserdu)

Stemline Therapeutics Inc.

  • Increased safety and efficacy
  • Fast Track
  • Priority Review

Human epidermal growth factor receptor 2 negative breast cancer (HER2- Breast Cancer); Metastatic breast cancer

Approval

  • Approved by the US FDA (Orserdu; January 27, 2023).242 [Note: US company is listed as Stemline Therapeutics, Inc]

 Forecasted revenue

  • Total global annual revenue forecasted to be $57 million by 2028.*

* Consensus forecasts for global revenue data were collected from GlobalData, Q4-2022, and are given in US dollars.
Data source: GlobalData Healthcare database.

Spotlight on Canada

This section includes a list of select medicines currently under review by Health Canada that may have a significant impact on future clinical practice and drug spending. Medicines included on this list may be new to Canada but have been approved in other jurisdictions.

Health Canada’s Drug and Health Product Submissions Under Review (SUR) Lists include biosimilars. Although they do not have improved safety and efficacy, their availability could have a potential impact on drug spending. The biosimilars under review, as of September 2022 are: aflibercept, bevacizumab, eculizumab, enoxaparin, pegfilgrastim, and trastuzumab. To date, there are no biosimilars for aflibercept or eculizumab. Their availability could have a potential impact on the costs set aside in drug budgets for the use of these therapies. Appendix A (Table A2) lists the biosimilars currently under review with Health Canada.

Table 7 highlights five new medicines currently on Health Canada’s Drug and Health Product SUR Lists that have a novel mechanism of action or have demonstrated improved safety and efficacy in clinical trials. Of the four medicines reported in the 2021 edition, all have since received market authorization from Health Canada.

The SUR Lists are publicly available sources that identify pharmaceutical and biologic drug submissions with new medicinal ingredients that have been accepted for review in Canada.

Table 7. Selected new medicines currently under review by Health Canada, 2022

Central Nervous System

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Masitinib mesylate

AB Science S.A.

  • Novel mechanism

Amyotrophic lateral sclerosis (ALS)

  • Add-on therapy
  • Rare or orphan designation
  • A selective tyrosine kinase inhibitor.
  • Administered orally, in combination with riluzole.

Clinical trials

  • In a survival analysis of patients previously randomized for a Phase III study, it was found to prolong survival by over 2 years as compared with placebo, in patients whose treatment started prior to severe impairment of functionality.243
  • An application has been filed with EMA for a conditional authorization.244 [Note: In 2018, the EMA refused authorization for ALS (Alsitek; April 18, 2018245), but according to the manufacturer’s information, issues identified by the EMA at that time have been addressed.]
  • There are other drugs in Phase II development for this indication but none with the same mechanism of action as Masitinib. 246 See Appendix A for additional information.

 Forecasted revenue

  • Forecasted annual global revenue unknown.

Hematological disorders

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Concizumab

Novo Nordisk Canada Inc

  • Novel mechanism
  • Breakthrough

Haemophilia B

  • Rare or orphan designation
  • Biologic medicine
  • A monoclonal, humanized IgG4 antibody specific for the Kunitz-2 domain of Tissue Factor Pathway Inhibitor (TFPI).247
  • Administered once daily subcutaneously as prophylactic therapy (regular treatment to prevent prolonged and spontaneous bleeding) for hemophilia.248

Clinical trials

  • Results of the Phase III trial showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean annualized bleeding rate (ABR) of 1.7 compared to 11.8 with no prophylaxis.249
  • It offers the potential for everyday protection for people living with haemophilia and provides an important potential addition, especially in the haemophilia B with inhibitor population who currently have limited treatment options. 250
  • There are other drugs in Phase II development for this indication but none with the same mechanism of action as concizumab. 251 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $177 million by 2028.*

Immunology

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Spesolimab

Boehringer Ingelheim (Canada) Ltd Ltee

  • Novel mechanism
  • Breakthrough
  • Priority Review

Generalized pustular psoriasis (GPP)

  • Biologic medicine
  • Rare or orphan designation
  • A humanized monoclonal antibody that acts as an interleukin (IL)-36 receptor antagonist.
  • It is administered intravenously.

Clinical trials

  • In the 12-week pivotal clinical trial, it was rapidly effective in the majority of patients within one week of its first intravenous infusion for patients suffering from generalized pustular psoriasis.252, 253
  • Generalized pustular psoriasis (GPP) is a rare, life-threatening condition. There are no approved options to help manage GPP flares.254, 255, 256
  • Approved by the US FDA (SPEVIGO; September 1, 2022) 257 and conditionally by the EMA (Spevigo; October 13, 2022).258
  • No other therapy was identified in Phase II development at this time.259

Forecasted revenue

  • Forecasted annual global revenue unknown.

Oncology

Medicine (Trade name) Company Indication(s)* Description and Key Attributes

Ciltacabtagene autoleucel

(Carvykti)

Janssen Inc

  • Increased safety and efficacy
  • Gene or cell therapy
  • Breakthrough
  • Priority Review

Relapsed or refractory multiple myeloma

  • Biologic medicine
  • Rare or orphan designation
  • Health Canada approved but not yet marketed as of February 9, 2023.
  • A chimeric antigen receptor (CAR) T-cell therapy that targets B-cell maturation antigen (BCMA) on the surface of cancer cells in B-cell malignancies, such as multiple myeloma. 260
  • It is administered intravenously.

Clinical trials

  • In a main study, a single infusion was effective at clearing cancer cells in patients with multiple myeloma that had returned and did not respond to three or more previous treatments. After one and a half years, about 84% of patients (95 out of 113) had a good response to the treatment and in 69% (78 out of 113) the signs of cancer had disappeared (complete response). It was not compared to another medicine in this study. These results were better than those seen in other studies of patients receiving standard treatments for multiple myeloma.261
  • It generally outperformed idecabtagene vicleucel (Abecma) in terms of efficacy in MM, but showed comparable adverse events.262
  • It showed superior efficacy compared with physician's choice of treatment, making it a promising new treatment option for patients with triple-class exposed relapsed or refractory multiple myeloma.263
  • Approved by the US FDA (Carvykti ; February 28, 2022)264 and by the EMA (Carvykti; June 17, 2022).265
  • There are other gene therapies for this indication in Phase II development at this time. 266 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $4.8 billion by 2028.*

Glofitamab

Hoffmann-La Roche Limited

  • Novel mechanism
  • Priority Review

Diffuse large B-cell lymphoma

  • Biologic medicine
  • Rare or orphan designation
  • It is a full-length, IgG-like bispecific CD20-CD3 with a unique 2:1 configuration that provides an extended half-life and superior CD20 binding.267
  • It is administered intravenously.

Clinical trials

  • Fixed-duration glofitamab (given for a fixed amount of time, and not taken until disease progression) generated a notable objective response rate and complete remission rate in patients with heavily pretreated, highly refractory large B-cell lymphoma.268, 269
  • It appears to be “a welcome addition to the treatment possibilities for patients with B-cell lymphomas who otherwise have limited therapeutic options.” 270
  • Under review by the EMA.271
  • There are other drugs in Phase II development for this indication but none with the same mechanism of action as glofitamab.272 See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $634 million by 2028.*

* Consensus forecasts for global revenue data were collected from GlobalData, Q4-2022, and are given in US dollars.
Health Canada’s Drug and Health Product Submissions Under Review Lists provide the therapeutic area for the medicine under review but do not specify the indication. The indication listed in Table 7 is based on the information about the medicine in the literature and/or approvals in other jurisdictions. When there is an aligned review, in some cases the indication was confirmed by the CADTH Reimbursement Review report.
Data source: GlobalData Healthcare database.

Emerging COVID-19 Therapies

This section of the Meds Pipeline Monitor includes an overview of new and existing pipeline medicines that are under clinical evaluation for indications related to the prevention and treatment of COVID-19. An analysis of global markets provides information on COVID-19 medicines in all phases of clinical trials and pre-registration.

Global markets

The COVID-19 drug pipeline has developed at unprecedented rates over the past 3 years. Published information confirming the safety and efficacy of the various treatments for COVID-19 is continuously evolving.

In addition to the wide variety of vaccines under development, many novel and repurposed medicines are currently being evaluated in clinical trials for their potential benefits in the treatment of COVID-19. These include antivirals, monoclonal antibodies, synthetic peptides and cell therapies.273

A breakdown of the COVID-19 pipeline vaccines and treatments by phase of clinical evaluation is shown in Figure 5. For this snapshot, data was extracted for medicines indicated for the treatment of COVID-19 with a development stage of Phase I, II, III, or pre-registration. These medicines are presented in three categories: vaccines, which are used to prevent infection of the novel coronavirus; treatments (new medicines), which are new medicines used for the prevention or reduction of some of the complications associated with COVID-19 (e.g., pneumonia or respiratory complications and hyperinflammation); and treatments (existing medicines), which are previously marketed medicines that have been repurposed to treat COVID-19 or its symptoms.

Brief Insights

The pipeline for COVID-19 medicines continues to grow, with clinical investigations of novel and existing drugs. The following are some of the most significant advancements in 2022:

  • In 2022, over 400 novel medicines were undergoing clinical evaluation for the treatment and prevention of COVID-19. This is a 10% increase from last year.
  • Advances in vaccine technology have allowed for rapid updates and approvals for existing vaccines to protect against new strains of COVID-19.
  • Bivalent vaccines that target the BA.4 and BA.5 subvariants of Omicron have been approved in many countries worldwide.
  • Nasal vaccines are a growing alternative approach to preventing the infection of COVID-19. In 2022, there were 20 nasal vaccines in various phases of the pipeline. Currently there are two nasal or inhaled vaccines approved in China and India.
  • More effective treatment options in the pipeline include oral antivirals and new monoclonal antibodies that have shown positive results in clinical trials.
  • Stem cell therapy and stem cell-derived organoid models are a growing new treatment option and research method for COVID-19. As of February 2023, there were 53 cell therapies undergoing clinical evaluation in various phases of the pipeline.

Source: GlobalData Healthcare Database (February, 2023); Health Canada (February, 2023).

Figure 5 illustrates the number of clinical trials for COVID-19 treatments and vaccines by latest development phase as of September 2022. The majority of vaccines in the pipeline (97%) are new medicines intended to prevent COVID-19 infection. Antivirals are the most common therapy used to treat COVID-19 symptoms, with 74% new medicines in the pipeline. Other treatment options include monoclonal antibodies, cell therapies and synthetic peptides, that have a larger percentage of redirected and repurposed medicines in the pipeline.

Figure 5. Number of medicines undergoing clinical evaluation for the prevention and treatment of COVID-19 by phase of development, 2022 Figure 5. Number of COVID-19 vaccines and treatments in the pipeline, by phase of clinical evaluation, 2020
Data source: GlobalData (accessed September 2022).
Figure description

This stacked bar graph illustrates the number of COVID-19 treatments and vaccines in the pipeline in 2022. Totals are given for vaccines and treatments, with separate totals for new treatments and repurposed or redirected treatments.

  Phase I Phase II Phase III Pre-registration

Vaccine

53

59

43

2

New treatment

73

159

59

1

Repurposed / Redirected treatment

32

33

34

2

Figure 6 breaks down the COVID-19 vaccines by mechanism of action and highest development phase.Footnote xiv Vaccines are categorized into various vaccine types based on their mechanism of action; for example, while live attenuated vaccines target the whole virus, protein subunit and recombinant vector vaccines target one specific part of the virus.

Figure 6. Distribution of COVID-19 vaccines by mechanism of action and phase of development, 2022 Distribution of COVID-19 vaccines by category and phase of clinical evaluation, 2020

Data source: GlobalData (accessed September 2021).

Figure description

A stacked bar graph gives the distribution of coronavirus vaccines in each phase of clinical evaluation by vaccine type, as of September 2022.

  Protein Subunit Vaccine Inactivated Vaccine mRNA Vaccine Recombinant Vector Vaccine DNA Vaccine Live Attenuated Vaccine Total

Phase I

20

2

7

10

12

2

53

Phase II

25

6

13

9

6

 0

59

Phase III

16

13

4

5

3

2

43

Pre-registration

1

 0

1

 0

 0

 0

2

Canada

COVID-19 continues to impact Canadians, with approximately 182,000 hospitalizations and over 52,000 deaths as of March 2023. Footnote xv Over 85% of Canadians have completed their primary series of vaccinations (two doses). Footnote xvi Health Canada’s most recently approved vaccines are bivalent vaccines that target two different strains of the virus. The updated Moderna Spikevax and Pfizer-BioNTech Comirnaty vaccines target the original SARS-CoV-2 virus as well as the Omicron BA.4 and BA.5 subvariants, which were known to be resistant to previous versions of the vaccines. The vaccines are produced using the same methods as previous COVID-19 vaccines, except that they contain two mRNA components instead of one, which allows them to target more than one strain of the virus.

Health Canada is prioritizing reviews of all COVID-19 vaccine submissions. As of March 2023, Health Canada has approved 6 vaccines, including two mRNA vaccines (Comirnaty and Spikevax), two viral vector-based vaccines (Jcovden and Vaxzevria), one plant-based vaccine (Covifenz), and one protein-based vaccine (Nuvaxovid). Table 8 provides the number of medicines approved by Health Canada for the prevention and treatment of COVID-19, while Table 9 gives the number of COVID-19 medicines under review with Health Canada as of March 2023.

Table 8. COVID-19 treatments and vaccines approved by Health Canada, 2022

Therapeutic area Applicant Medicinal ingredient(s) Outcome of application Date of decision/outcome

Antivirals for systemic use

Veklury
Gilead Sciences Canada Inc.

Remdesivir (solution for injection)

Approved under:

Food and Drug Regulations; Notice of Compliance issued under the NOC/c Guidance

27-Jul-20

Expanded indication:

Food and Drug Regulations; authorized

22-Apr-22

Antivirals for systemic use

Paxlovid
Pfizer Canada ULC

Nirmatrelvir and ritonavir (tablets for oral administration)

Approved under:

Food and Drug Regulations; authorized with terms and conditions

17-Jan-22

Immune sera and immunoglobulins

Evusheld
AstraZeneca Canada Inc.

Cilgavimab and tixagevimab solution for injection

Approved under:

Food and Drug Regulations; authorized with terms and conditions

14-Apr-22

Expanded indication:

Food and Drug Regulations; authorized

18-Oct-22

Immune sera and immunoglobulins

Casirivimab and imdevimab
Hoffmann-La Roche Ltd

Casirivimab and imdevimab (solution for injection)

Approved under:

Interim order;* authorized with terms and conditions

09-Jun-21

Immune sera and immunoglobulins

Bamlanivimab
Eli Lilly Canada Inc.

Bamlanivimab (solution for injection)

Approved under:

Interim order;* authorized with terms and conditions

20-Nov-20

Immune sera and immunoglobulins

Sotrovimab
GlaxoSmithKline Inc.

Sotrovimab (solution for injection)

Approved under:

Interim order;* authorized with terms and conditions

30-Jul-21

Immune sera and immunoglobulins

Actemra
Hoffmann-La Roche Ltd

Tocilizumab solution for injection

Expanded indication:

Food and Drug Regulations; authorized

13-Oct-22

Vaccines

Covifenz
Medicago Inc

Virus-like particles of SARS-CoV-2 spike protein

Approved under:

Food and Drug Regulations; authorized with terms and conditions

24-Feb-22

Vaccines

Nuvaxovid
Novavax Inc.

SARS-CoV-2 recombinant spike protein

Approved under:

Adolescent dose (ages 12-17 years)
Food and Drug Regulations

06-Dec-22

First booster dose
Food and Drug Regulations; authorized with terms and conditions

17-Nov-22

Food and Drug Regulations; authorized with terms and conditions

17-Feb-22

Vaccines

Vaxzevria
AstraZeneca Canada Inc.

ChAdOx1-S [recombinant]
(solution for injection)

Approved under:
Food and Drug Regulations; authorized with terms and conditions

19-Nov-21

Interim order*

26-Feb-21

Vaccines

Comirnaty
BioNTech Manufacturing GmbH

Tozinameran [mRNA vaccine, BNT162b2] (suspension for injection)

Approved under:
Bivalent booster (ages 5-11 years)
Food and Drug Regulations; authorized with terms and conditions

09-Dec-22

Bivalent booster (ages 12 years and over)
Food and Drug Regulations; authorized with terms and conditions

07-Oct-22

Pediatric indication (ages 6 months-5 years)
Food and Drug Regulations; authorized with terms and conditions

09-Sep-22

First booster dose (ages 5-11 years)
Food and Drug Regulations; authorized with terms and conditions

19-Aug-22

First booster dose (ages 16-17 years)
Food and Drug Regulations; authorized with terms and conditions

01-Jun-22

Food and Drug Regulations; pediatric indication (ages 5-11)

19-Nov-21

Food and Drug Regulations; first booster dose

09-Nov-21

Food and Drug Regulations; authorized with terms and conditions

16-Sept-21

Interim order;* pediatric indication (ages 12-15)

05-May-21

Interim order*

09-Dec-20

Vaccines

Spikevax
Moderna TX, Inc.

Elasomeran
(suspension for injection)

Approved under:

First booster dose (ages 12-17 years)
Food and Drug Regulations; authorized with terms and conditions

12-Jan-23

Bivalent booster (ages 18 years and over)
Food and Drug Regulations; authorized with terms and conditions

03-Nov-22

Bivalent booster (ages 18 years and over)
Food and Drug Regulations; authorized with terms and conditions

01-Sep-22

Pediatric indication (ages 6 months-5 years)
Food and Drug Regulations; authorized with terms and conditions

14-Jul-22

Pediatric indication (ages 6-11 years)
Food and Drug Regulations; authorized with terms and conditions

17-Mar-22

First booster dose
Food and Drug Regulations; authorized with terms and conditions

12-Nov-21

Food and Drug Regulations; authorized with terms and conditions

16-Sept-21

Interim order;* pediatric indication (ages 12-17)

27-Aug-21

Interim order*

23-Dec-20

Vaccines

Jcovden
Janssen Inc.

AD26.COV2.S [recombinant]
(suspension for injection)

Approved under:

First booster dose
Food and Drug Regulations; authorized with terms and conditions

11-May-22

Food and Drug Regulations; authorized with terms and conditions

23-Nov-21

Interim order*

05-Mar-21

Vaccines

Covishield
Verity Pharmaceuticals Inc/Serum Institute of India (in partnership with AstraZeneca Canada Inc)

ChAdOx1-S (recombinant)

Approved under:

Interim order;* authorized with terms and conditions

26-Feb-2021 (expired 16-Sept-21)

* The Interim Order Respecting the Importation, Sale and Advertising of Drugs for Use in Relation to COVID-19, approved on May 23, 2020, introduced an alternate pathway to facilitate clinical trials for potential COVID-19 drugs and medicinal devices, while upholding strong patient safety requirements and validity of trial data.

Data source: Drug and vaccine authorizations for COVID-19: List of authorized drugs, vaccines and expanded indications (accessed March 2023):
https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry/drugs-vaccines-treatments/authorization/list-drugs.html

Table 9. COVID-19 treatments and vaccines under review by Health Canada, as of March 2023

Therapeutic area Applicant Medicinal ingredient(s) Date submission accepted

Vaccines

Pfizer Canada ULC/ BioNTech SE

Tozinameran

Feb-22

Vaccines - Booster dose

AstraZeneca Canada Inc.

 [ChAdOx1-S; [recombinant]

Dec-21

Vaccines - Booster dose

Janssen Inc.

Ad26.COV2.S

Dec-21

Vaccines - Pediatric dose (ages 6-11)

ModernaTX, Inc.

Elasomeran

Nov-21

Vaccines

Medicago Inc.

Coronavirus-like particle [CoVLP]

Aug-21

Vaccines

Novavax Inc.

NVX-CoV2373

Aug-21

Vaccines

Sanofi Pasteur Ltd

SARS-CoV-2 prefusion spike delta TM protein [recombinant]

Jul-21

Vaccines

Vaccigen Ltd

Whole virion inactivated coronavirus

Jul-21

Immune sera and immunoglobulins

AstraZeneca Canada Inc.

Cilgavimab, tixagevimab

Nov-21

Immune sera and immunoglobulins

Celltrion HealthCare Co. Ltd

Regdanvimab

May-21

Immune sera and immunoglobulins

Eli Lilly Canada Inc.

Bamlanivimab*

Jun-21

Immune sera and immunoglobulins

Hoffmann-La Roche Ltd

Casirivimab, imdevimab*

Sept-21

Immune sera and immunoglobulins

GlaxoSmithKline Inc.

Sotrovimab*

Oct-21

Immune sera and immunoglobulins

Eli Lilly Canada Inc.

Etesevimab

Sept-21

Immunosuppressants

Eli Lilly Canada Inc

Baricitinib

Sep-21

Antivirals for systemic use

Gilead Sciences Canada Inc

Remdesivir

Apr-21

Antivirals for systemic use

Dr Reddys Laboratories Ltd

Favipiravir

Sept-21

Antivirals for systemic use

Merck Canada Inc

Molnupiravir

Aug-21

Ceased reviews

Immune sera and immunoglobulins

CytoDyn Inc.

Leronlimab

Mar-21

(Expired)

Antigout preparations

Pendopharm Division of Pharmascience Inc

Colchicine

Jan-21
07-Jun-21 cancelled by sponsor

Other nervous system drugs

Sanotize Research & Development Corp.

Nitric oxide

Jun-21
01-Sep-21 cancelled by sponsor

* The applicant has filed a new drug submission under the Food and Drug Regulations to transition this product from the interim order. The product continues to be approved for sale in Canada during this transition period.

Data source: Drug and vaccine authorizations for COVID-19: List of applications received, Health Canada (accessed March 2023): https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry/drugs-vaccines-treatments/authorization/applications.html

Appendix A

Table A1: Biosimilars in Phase III (based on data extract from 2022-09)

Medicine Reference product (Manufacturer) Other biosimilars (Y/N) Manufacturers developing biosimilars Indication(s)

Adalimumab

 

Humira

(Abbvie Corp)

 

Y

Enzene Biosciences Ltd

Ankylosing spondylitis (Bekhterev's disease)

Wuhan Institute of Biological Products Co Ltd

Plaque psoriasis (psoriasis vulgaris)

Aflibercept

 

Eylea

(Bayer Inc)

 

N

Amgen Inc

Alteogen Inc

Alvotech SA

Celltrion Inc

Formycon AG

Hexal AG

Johnson & Johnson

Sam Chun Dang Pharm Co Ltd

Samsung Bioepis Co Ltd

Wet (neovascular/exudative) macular degeneration

Bevacizumab

Avastin

(Hoffmann-La Roche Limited)

Y

Aurobindo Pharma Ltd

Prestige BioPharma Ltd

SinoCelltech Group Ltd

 

Centrion

Non-small cell lung cancer

 

 

 

N/A

Cetuximab

Erbitux

N

Ampo Biotechnology Inc

Cinnagen Co

Enzene Biosciences Ltd

R-Pharm

Head and neck cancer squamous cell carcinoma; Lip cancer; Locally recurrent or locoregional solid malignancies; Oral cavity (mouth) cancer; Pharyngeal neoplasm; Metastatic colorectal cancer

Darbepoetin alfa

Aranesp

(Amgen Canada Inc)

N

Biocad

 

Anemia in chronic kidney disease (renal anemia)

Denosumab

 

 

Prolia / Xgeva

(Amgen Canada Inc)

N

Mabwell Shanghai Bioscience Co Ltd

Diabetes

Celltrion Inc

Eden Biologics Inc

Fresenius Kabi SwissBioSim GmbH

Gedeon Richter Plc

Mabxience Holding SL

Samsung Bioepis Co Ltd

Sandoz International GmbH

Teva Pharmaceutical Industries Ltd

Postmenopausal osteoporosis

Sandoz International GmbH

Bone metastasis; Giant cell tumour of bone

Eculizumab

Soliris

(Alexion Pharma GmbH)

N

Amgen Inc

Samsung Bioepis Co Ltd

Paroxysmal nocturnal hemoglobinuria

Enoxaparin

Lovenox

(Sanofi-Aventis Canada Inc)

Y

Baxter Corporation

Fresenius Kabi Canada Ltd

N/A

Etanercept

Enbrel

(Immunex Corporation)

Y

Mycenax Biotech Inc

 

Rheumatoid arthritis

Follitropin alfa

Gonal-F / Pergoveris

(EMD Serono, a division of EMD Inc., Canada)

N

Amega Biotech

Female infertility

Golimumab

Simponi

(Janssen Inc)

N

Bio-Thera Solutions Ltd

 

Psoriatic arthritis

Liraglutide

Victoza / Saxenda

(Novo Nordisk Canada Inc)

N

Shanghai Fosun Pharmaceutical (Group) Co Ltd

Obesity

Ocrelizumab

 

Ocrevus

(Hoffmann-La Roche Limited)

N

Cinnagen Co

Relapsing remitting multiple sclerosis (RRMS)

Omalizumab

Xolair

(Novartis Pharmaceuticals Canada Inc)

 

N

Synermore Biologics Co Ltd

Allergic asthma

Celltrion Inc

Synermore Biologics Co Ltd

Teva Pharmaceutical Industries Ltd

Chronic urticaria or hives

Synermore Biologics Co Ltd

Nasal polyps (nasal polyposis); Rhinosinusitis

Pegfilgrastim

Neulasta

(Amgen Canada Inc)

Y

Lupin Pharma Canada Limited

Nora Pharma Inc

Febrile neutropenia

Pertuzumab

Perjeta

(Hoffmann-La Roche Limited)

N

Zydus Lifesciences Ltd

Human epidermal growth factor receptor 2-positive breast cancer (HER2+ breast cancer)

Ranibizumab

Lucentis

(Novartis Pharmaceuticals Canada Inc)

 

Y*

Aurobindo Pharma Ltd

Enzene Biosciences Ltd

Generium

Jecho Biopharmaceuticals Co Ltd

Lupin Ltd

PharmaResearch BIO Co Ltd

Wet (neovascular/exudative) macular degeneration

Romiplostim

Nplate

(Amgen Canada Inc)

N

Generium

 

Idiopathic thrombocytopenic purpura (immune thrombocytopenic purpura)

Tenecteplase

TNKase

(Hoffmann-La Roche Limited)

N

Hetero Drugs Ltd

 

Myocardial infarction

Trastuzumab

Herceptin

(Hoffmann-La Roche Limited)

Y

Aprogen Inc

Hetero Drugs Ltd

Tanvex BioPharma Inc

Prestige BioPharma Ltd. (HC)

Human epidermal growth factor receptor 2-positive breast cancer (HER2+ Breast Cancer)

Ustekinumab

Stelara

(Janssen Inc)

N

DM Bio Ltd

Samsung Bioepis Co Ltd

Psoriatic arthritis

Amgen Inc

Alvotech SA

Bio-Thera Solutions Ltd

Celltrion Inc

DM Bio Ltd

Formycon AG

Samsung Bioepis Co Ltd

Plaque psoriasis (psoriasis vulgaris)

DM Bio Ltd

Crohn's disease (regional enteritis); Ulcerative colitis

JiangSu Qyuns Therapeutics Co Ltd

Inflammatory bowel disease

* Approved but not marketed as of December 5, 2022.
 Biosimilar currently under review by Health Canada.

Table A2: New drug submissions under review by Health Canada (February 2023)

Medicinal ingredient Therapeutic area Manufacturer Date submission accepted for review

Aflibercept

Ophthalmologicals

BGP Pharma ULC

2022-05

Bevacizumab

Antineoplastic agents

Celltrion Healthcare Co Ltd


2022-03

Eculizumab

Immunosuppressants

Amgen Canada Inc

2022-07

Enoxaparin sodium

Antithrombotic agents

Baxter Corporation

2022-09

Pegfilgrastim

Immunostimulants

Lupin Pharma Canada Limited

2022-05

Pegfilgrastim

Immunostimulants

Nora Pharma Inc


2022-05

Ranibizumab

Ophthalmologicals

Teva Canada Limited

2022-12

Trastuzumab

Antineoplastic agents

Prestige BioPharma Ltd.

2021-08

 

Table A3: Drugs in Phase II for indications targeted by pipeline candidates

Pipeline candidate Indication(s) Drugs in Phase II and mechanism of action (MOA)*

Abelacimab

Deep vein thrombosis (DVT); Pulmonary embolism

  • Undisclosed MOA (TF-0023, Zifa-01).

There are no other drugs for this indication with the same MOA as abelacimab (a dual Factor XI and Factor Xia inhibitor) in Phase II development at this time.

Aprocitentan

and

Firibastat

Resistant hypertension

  • Angiotensinogen inhibitor (evazarsen, tonlamarsen);
  • Atrial natriuretic peptide receptor 1 agonist (PL-3994);
  • Cytochrome P450 11B2 mitochondrial inhibitor (baxdrostat).

There are no other drugs for this indication with the same MOA as aprocitentan (a dual endothelin-receptor antagonist) or firibistat (a centrally-acting, aminopeptidase A inhibitor) in Phase II development at this time.

Bemarituzumab

and

Zolbetuximab

Adenocarcinoma of the gastroesophageal junction; Gastric cancer; bladder cancer; Gastroesophageal (GE) junction carcinomas

  • Angiopoietin 2 inhibitor (BI-836880);
  • C-C chemokine receptor Type 8 antagonist (BMS-986340, S-531011);
  • Cell therapy (RAPA-201);
  • Claudin-18 inhibitor (BNT-141, LM-302);
  • Cytotoxic to cells expressing:
  • Claudin-18 (CT-041, SOT-102);
  • Epidermal growth factor receptor (AFM-24I, cetuximab, MRG-003);
  • Intercellular adhesion molecule 1 (gebasaxturev);
  • Receptor tyrosine protein kinase ERBB 2 (cinrebafusp alfa, DP-303c);
  • Stabilin 1 (bexmarilimab);
  • Telomerase reverse transcriptase (suratadenoturev);
  • Delta like protein 4 inhibitor (navicixizumab);
  • Deoxyuridine 5' triphosphate nucleotidohydrolase mitochondrial inhibitor (TAS-114);
  • Dickkopf related protein 1 inhibitor (DKN-01);
  • DNA synthesis inhibitor (doxorubicin);
  • DNA topoisomerase i inhibitor (irinotecan);
  • Epidermal growth factor receptor antagonist (GC-1118A, MCLA-129);
  • Fc fragment of IgG low affinity iii receptor agonist (CYNK-101);
  • Fibroblast growth factor receptor inhibitor (MAX-4);
  • Gremlin 1 inhibitor (UCB-6114);
  • Histone lysine N methyltransferase EZH2 inhibitor (CPI-0209);
  • Interleukin 2 receptor agonist (SAR-444245);
  • Interleukin 7 receptor subunit alpha agonist (efineptakin alfa);
  • Leukocyte immunoglobulin like receptor subfamily B member 1 antagonist (SAR-444881);
  • Leukocyte surface antigen CD47 inhibitors (AO-176, ligufalimab);
  • Low affinity immunoglobulin gamma Fc region receptor II-b antagonist (BI-1607);
  • Lymphocyte activation gene 3 protein inhibitor (miptenalimab);
  • Mitogen activated protein kinase 1 inhibitors (NMBS-2, ulixertinib);
  • Nuclear receptor ROR Gamma agonist (cintirorgon);
  • 5' nucleotidase inhibitor (uliledlimab);
  • Programmed cell death 1 ligand 1 inhibitor (ezabenlimab, HB-0036);
  • Serine/threonine protein kinase ATR inhibitor (ceralasertib);
  • Smoothened homolog antagonist (taladegib);
  • T cell immunoreceptor with Ig and ITIM domains antagonist (etigilimab);
  • Tumor necrosis factor receptor superfamily member 5 agonist (sotigalimab);
  • Tumor necrosis factor receptor superfamily member 18 agonist (ragifilimab);
  • Vaccines (IMU-131, ombipepimut-s);
  • Vascular endothelial growth factor inhibitor (KH-903);
  • Zinc transporter ZIP6 inhibitor (ladiratuzumab vedotin);
  • Others with undisclosed mechanism of action (ecubectedin, GEN-001, VE-800).

There is another drug (MAX-4) with a similar MOA to bemarituzumab (a fibroblast growth factor receptor inhibitor) and others (BNT-141, LM-302) with a similar MOA to zolbetuximab (a claudin-18 inhibitor) in Phase II development at this time.

Ciltacabtagene autoleucel*

and

Iberdomide hydrochloride

Refractory multiple myeloma; Relapsed multiple myeloma

  • Activin receptor Type 1 antagonist (INCB-00928);
  • ADP ribosyl cyclase/cyclic ADP ribose hydrolase 1 inhibitors (BHV-1100, mezagitamab);
  • Arginase 1 inhibitor (numidargistat);
  • ATP dependent Clp protease proteolytic subunit mitochondrial activator (ONC-201);
  • B-cell lymphoma 2 inhibitor (APG-2575, BGB-11417);
  • CD3 agonist (ABBV-383, cevostamab, HPN-217, ISB-1342, linvoseltamab, REGN-5459, talquetamab);
  • Cell therapy (various, ACP- 001, Anti-BCMA/GPRC5D CAR-T, ARI-0002h, CAR-BCMA, ECT-001, GDA-201, NEXI-002, NK Cell Multiple Myeloma, RAPA-201, VAX-DC);
  • CREB binding protein inhibitor (inobrodib);
  • Cytotoxic to cells expressing:
  • ADP ribosyl cyclase/cyclic ADP ribose hydrolase 1 (GEN-3014, modakafusp alfa, STI-6129);
  • Carcinoembryonic antigen related cell adhesion molecule 8 (TLX-66);
  • Kappa myeloma antigen (MDX-1097);
  • Membrane cofactor protein (oncolytic virus);
  • Tumor necrosis factor receptor superfamily member 17 (ALLO-605, CC-99712, HDP-101);
  • Dickkopf related protein 1 inhibitor (BHQ-880);
  • DNA repair protein RAD51 homolog 1 inhibitor (CYT-0851);
  • DNA synthesis inhibitor (doxorubicin);
  • Dual specificity mitogen activated protein kinase kinase 1 inhibitor (trametinib dimethyl sulfoxide + uprosertib);
  • Exportin 1 inhibitor (eltanexor);
  • Fibroblast growth factor receptor 1 inhibitor (ABSK-091);
  • Gene-modified cell therapy (various, Descartes-08, Descartes-11, Descartes-25, GDT-002, KJC-2111, letetresgene autoleucel, OPC-415, orvacabtagene autoleucel, PBCAR-269A, PHE-885, SENL-302, zevorcabtagene autoleucel);
  • Heparanase inhibitor (roneparstat);
  • Hepatocyte growth factor inhibitor (MP-0250);
  • Histone deacetylase 6 inhibitor (ricolinostat);
  • Interleukin 2 receptor subunit alpha agonist (ANV-419);
  • Interleukin 15 receptor subunit alpha agonist (NKTR-255);
  • Interleukin 18 inhibitor (AVTX-007);
  • Killer cell immunoglobulin like receptor 2DL1 Antagonist (lirilumab);
  • Leukocyte surface antigen CD47 inhibitor (AO-176, PF-07901801);
  • Phosphatidylinositol 4,5 bisphosphate 3 kinase catalytic subunit beta isoform inhibitor (GSK-2636771);
  • Programmed cell death protein 1 antagonist (pidilizumab);
  • 20s proteasome inhibitor (CX-13608);
  • Protein cereblon activator (CFT-7455, mezigdomide);
  • Protein S100 A9 inhibitor (tasquinimod);
  • RAC alpha serine/threonine protein kinase inhibitor (uprosertib);
  • Small ubiquitin related modifier 2 inhibitor (subasumstat);
  • T cell immunoreceptor with Ig and ITIM domains antagonists (belrestotug, BMS-986207);
  • TGF beta receptor Type 1 inhibitor (vactosertib);
  • Vaccines (biropepimut-S, GVAX Multiple Myeloma Vaccine, ImMucin, IO-103, PVX-410);
  • Wee1 like protein kinase inhibitor (adavosertib);
  • Others with undisclosed MOA (CB-103 , imifoplatin, iopofosine i-131, KES-0001).

There are other drugs (CFT-7455, mezigdomide) for this indication with a similar MOA to Iberdomide (a protein cereblon activator) and other gene therapies like ciltacabtagene in Phase II development at this time.

Concizumab*

Haemophilia B

  • Coagulation factor VII replacement (OPK-88005);
  • Coagulation factor IX activator (lanacogene vosiparvovec, verbrinacogene setparvovec);
  • Coagulation factor IX replacement (dalcinonacog alfa LA);
  • Coagulation factor X activator (STSP-0601);
  • Vitamin K dependent protein C inhibitor (SerpinPC).

There are no other drugs for this indication with the same MOA as concizumab (a tissue factor pathway inhibitor) in Phase II development at this time.

Evobrutinib

Relapsing multiple sclerosis (RMS); Secondary progressive multiple sclerosis (SPMS)

  • ACE inhibitor (lisinopril);
  • CD40 ligand inhibitor (SAR-441344);
  • Cell therapy (ATA-188, ATA-190, CLS-12311, NG-1);
  • Combination (ASA and dimethyl fumarate);integrin alpha 4 inhibitor (ATL-1102);
  • Lysine specific histone demethylase 1A inhibitor (vafidemstat);repulsive guidance molecule A inhibitor (elezanumab);
  • Serine/threonine protein kinase Sgk2 activator (OCS-05);
  • Sphingosine 1-phosphate receptor 1 antagonist (amiselimod);
  • T cell surface glycoprotein CD3 Epsilon chain antagonist (foralumab);
  • Other with an undisclosed MOA (IB-MS, IMCY-0141, MP-101, RNS-60, smderpept, temelimab , WP-1303).

There are no other drugs for this indication with the same MOA as evobrutinib (a selective, CNS penetrant immunomodulator that irreversibly blocks BTK) in Phase II development at this time.

FCR001

Kidney transplant rejection

  • Antibody (LIS-1);
  • CD40 ligand inhibitor (tegoprubart);
  • Cell therapies (MDR-102, MIC-Lx, TX-200);
  • T cell specific surface glycoprotein CD28 antagonists (FR-104, lulizumab pegol);
  • T cell surface antigen CD2 inhibitor (siplizumab);
  • T lymphocyte activation antigen CD80 inhibitor (belatacept).

There are other cell therapies like FCR001 for this indication in Phase II development at this time.

Firibastat

 

Refer to information under “aprocitentan”

Garadacimab

Hereditary angioedema (HAE) (C1 esterase inhibitor [C1-INH] deficiency

  • Complement C1s subcomponent inhibitor (C1 esterase inhibitor);
  • Gene therapy (BMN-331);
  • Plasma kallikrein inhibitor (KVD-824, NTLA-2002).

There are no other drugs for this indication with the same MOA as garadacimab (an immunoglobulin G4 monoclonal antibody that interferes with FXIIa-mediated coagulation) in Phase II development at this time.

Glofitamab

B-cell lymphomas

  • CTP synthase 1 inhibitor (STP-938);
  • Cyclin dependent kinase 2 inhibitor (fadraciclib);
  • Gene-modified cell therapy (CD19/70 Bi-specific CAR-T cell therapy
  • Glucocorticoid receptor agonist (dexamethasone);
  • 2 oxoglutarate dehydrogenase inhibitor (devimistat).

There are no other drugs for this indication with the same MOA as glofitamab (a CD20xCD3 T-cell engaging bispecific antibody) in Phase II development at this time.

Iberdomide hydrochloride

 

Refer to information under “ciltacabtagene”

Imetelstat

and

Navitoclax

 

 

Myelodysplastic dyndrome; Post-rssential thrombocythemia myelofibrosis (post-ET MF); Post-polycythemia vera myelofibrosis (PPV-MF)

  • Activin receptor type 1 antagonist (INCB-00928);
  • Baculoviral IAP repeat containing protein 2 inhibitor (LCL-161);
  • Bromodomain containing protein 2 inhibitor (ABBV-744, BMS-986158);
  • E3 ubiquitin protein ligase Mdm2 inhibitor (siremadlin);
  • Glycogen synthase kinase 3 beta inhibitor (elraglusib);
  • Hemojuvelin inhibitor (DISC-0974);
  • Histone deacetylase 1 inhibitor (pracinostat);
  • Lysine specific histone demethylase 1A inhibitor (bomedemstat);
  • Lysyl oxidase homolog 2 inhibitor (GB-2064, PXS-5505A);
  • Serine/threonine protein kinase Pim 1 inhibitor (TP-3654);
  • Tyrosine protein kinase BTK inhibitor (TL-895);
  • Tyrosine protein kinase JAK2 inhibitor (ilginatinib);
  • Vaccine (Triplex);
  • Other with undisclosed MOA (INCB-57643).

There are no other drugs for this indication with the same MOA as imetelstat (a telomerase inhibitor) or navitoclax (an antagonist of a subset of the B-cell leukemia 2 family of proteins) in Phase II development at this time.

Masitinib*

and

Reldesemtiv

Amyotrophic Lateral Sclerosis (ALS)

  • Anti-apoptosis Stressin-1 peptide (IPL-344);
  • Apoptosis regulator BAX inhibitor (GM-6);
  • Arachidonate 15 lipoxygenase inhibitor (PTC-857);
  • Ataxin 2 inhibitor (ION-541);
  • CD40 ligand inhibitor (tegoprubart);
  • Cell therapy (astrorx, COYA-101, NG-1, RAPA-501);
  • Coagulation factor V inhibitor (3K3A-APC);
  • Combinations (acamprosate + baclofen, celecoxib + ciprofloxacin, nicotinamide riboside + pterostilbene);
  • Fibroblast growth factor receptor 1 agonist (FGF-1);
  • Glial cell line derived neurotrophic factor activator (gene therapy);
  • Guanine nucleotide exchange c9orf72 inhibitor (WVE-004);
  • Ikappa B kinase inhibitor (OP-101);
  • Macrophage colony stimulating factor 1 receptor antagonist (sotuletinib);
  • 1-phosphatidylinositol-3-phosphate-5-kinase inhibitor (apilimod);
  • Receptor interacting serine/threonine protein kinase 1 inhibitor (DNL-788);
  • Reverse transcriptase inhibitor (censavudine);
  • Reversible redox cofactor (EPI-589);
  • Superoxide dismutase activator (AP-101);
  • Other with an undisclosed MOA (MP-101, NP-001, RNS-60, TM-700).

There are no other drugs for this indication with the same MOA as masitinib (a selective tyrosine kinase inhibitor) or reldesemtiv (a fast skeletal muscle troponin activator) or in Phase II development at this time.

Navitoclax dihydrochloride

 

Refer to information under “imetelstat”

Obicetrapib

 

Dyslipidemia; Heterozygous familial hypercholesterolemia (heFH);

Atherosclerosis

  • Angiopoietin related protein inhibitor (AROANG-3, LY-3561774);
  • Apolipoprotein A inhibitor (olpasiran);
  • Dual inhibitor of cholesterol and triglyceride synthesis (gemcabene);
  • Geranylgeranyl pyrophosphate synthase inhibitor (antroquinonol);
  • Low density lipoprotein receptor agonist (AEM-28);
  • Proprotein convertase subtilisin/kexin type 9 inhibitor (AZD-8233, cepadacursen, MK-0616, NN-6434);
  • Thyroid hormone receptor beta agonist (VK-2809);
  • Toll like receptor 2 and 4 antagonist (VB-201);
  • Other with an undisclosed MOA (DWJ-1506, DWJ-1507, PC-mab).

There are no other drugs for this indication with the same MOA as obicetrapib (a CETP inhibitor) in Phase II development at this time.

Pegcetacoplan*

Paroxysmal Nocturnal Hemoglobinuria

  • Complement C5 inhibitor (tesidolumab);
  • Complement factor D inhibitor (ALXN-2050, BCX-9930).

There are no other drugs for this indication with the same MOA as pegcetacoplan (a complement C3 and fragment C3b inhibitor) in Phase II development at this time.

Relacorilant

Epithelial ovarian cancer

  • Arginase 1 inhibitor (numidargistat);
  • Bromodomain containing protein inhibitor (ff-21101);
  • Cd3 agonist (hpn-536, ubamatamab);
  • Cell membrane disruptor (at-101);
  • Cell therapy (avova-1);
  • Cellular tumor antigen p53 activator (kevetrin);
  • Cyclin dependent kinase 2 inhibitor (ebvaciclib, pf-07104091);
  • Cytotoxic to cells expressing:
  • Cd40 ligand (load-703);
  • Folate receptor alpha (farletuzumab ecteribulin);
  • Membrane cofactor protein (oncolytic virus to target cd46 and slc5a5 for oncology);
  • Mucin 16 (regn-5668);
  • Receptor tyrosine protein kinase erbb 2 (dp-303c);
  • Trophoblast glycoprotein (naptumomab estafenatox);
  • Delta like protein 4 inhibitor (navicixizumab);
  • Dickkopf related protein 1 inhibitor (dkn-01);
  • Dna topoisomerase i inhibitor (nlg-207);
  • Epidermal growth factor receptor antagonist (fmab-2
  • Focal adhesion kinase inhibitor (defactinib, in-10018);
  • Gene-modified cell therapy (various);
  • Granulocyte macrophage colony stimulating factor receptor subunit alpha agonist (oncos-102);
  • Heat shock protein 90 inhibitor (ganetespib);
  • Interleukin 12 subunit alpha activator (gen-1);
  • Leukocyte surface antigen cd47 inhibitor (ao-176, ligufalimab, pf-07901801);
  • Mitogen activated protein kinase 14 inhibitor (ralimetinib mesylate);
  • 5' nucleotidase inhibitor (uliledlimab);
  • 2'-5' oligoadenylate synthetase activator (poly-iclc);
  • Ornithine decarboxylase inhibitor (amxt-1501 + eflornithine);
  • Poly [adp ribose] polymerase 1 inhibitor (ceralasertib + olaparib);
  • Retinoic acid receptor agonist (fenretinide);
  • Serine/threonine protein kinase atr inhibitor (berzosertib);
  • Serine/threonine protein kinase chk1 inhibitor (esp-01);
  • Serine/threonine protein kinase mtor inhibitor (sapanisertib);
  • T cell immunoreceptor with ig and itim domains antagonist (bms-986207, etigilimab);
  • Transmembrane protein pvrig antagonist (com-701);
  • Tubulin inhibitor (docetaxel);
  • Tumor necrosis factor receptor superfamily member 5 agonist (sotigalimab);
  • Vaccines (maveropepimut-s, modified vaccinia ankara vaccine, ombipepimut-s, uv-1);
  • Wee1 like protein kinase inhibitor (znc-3);
  • Other with an undisclosed MOA (ecubectedin).

There are no other drugs for this indication with the same MOA as relacorilant (a glucocorticoid receptor antagonist) in phase ii development at this time.

Reldesemtiv

 

Refer to information under “masitinib”

REC-2282

Neurofibromatoses Type II (NF2)

Drugs in Phase II for this indication and their MOA:

  • 26s proteasome inhibitor (bortezomib).

There is no other drug for this indication with the same MOA as REC-2282 (a pan-histone deacetylase inhibitor) in Phase II development at this time.

Resmetirom

Non-alcoholic steatohepatitis (NASH); Non-alcoholic fatty liver disease (NAFLD)

  • Acetyl coa carboxylase inhibitor (clesacostat, firsocostat);
  • Adenosine monophosphate activated protein kinase activator (PXL-770);
  • Adenosine receptor A3 antagonist (LJ-2698);
  • Androgen receptor agonist (LPCN-1144);
  • Arachidonate 5 lipoxygenase inhibitor (tipelukast);
  • 17-Beta hydroxysteroid dehydrogenase 13 inhibitor (ARO-HSD);
  • Beta Klotho activator (BOS-580, NN-9499);
  • Bile acid receptor agonist (EDP-305, EYP-001, HPG-1860, TERN-101, tropifexor);
  • C-C motif chemokine 24 inhibitor (CM-101);
  • Cell therapy (hepastem);
  • Corticosteroid 11 beta dehydrogenase isozyme 1 inhibitor (AZD-4017);
  • Diacylglycerol O acyltransferase 2 inhibitor (ervogastat, ION-224);
  • Eotaxin inhibitor (bertilimumab);
  • Fibroblast growth factor receptor 1 agonist (efruxifermin, MK-3655);
  • Gastric inhibitory polypeptide receptor agonist (HM-15211);
  • Glucagon-like peptide 1 activator (BI-456906, efinopegdutide);
  • Ketohexokinase inhibitor (PF-06835919);
  • Lipopolysaccharide inhibitor (IMM-124E);
  • Mineralocorticoid receptor antagonist (apararenone);
  • Mitochondrial pyruvate carrier inhibitor (PXL-065);
  • Mitogen activated protein kinase 5 inhibitor (selonsertib);
  • NADPH oxidase inhibitor (APX-115);
  • Peptidyl prolyl cis trans isomerase A inhibitor (rencofilstat);
  • Protein glutamine gamma glutamyltransferase 2 inhibitor (ZED-1227);
  • Serpin H1 inhibitor (BMS-986263);
  • Sodium/glucose cotransporter 1 inhibitor (licogliflozin);
  • T cell surface glycoprotein CD3 Epsilon chain antagonist (foralumab);
  • Thyroid hormone receptor beta agonist (ASC-41, TERN-501, VK-2809);
  • Tyrosine protein kinase receptor UFO inhibitor (bemcentinib);
  • Combinations (cenicriviroc + tropifexor, cilofexor + firsocostat, clesacostat + ervogastat, leucine + metformin + sildenafil);
  • Other with an undisclosed MOA (ADTP-02, AXA-1125, B-105, epeleuton, gemcabene, HTD-1801, HU-6, icosabutate, INA-010, LM-011, MBK-002, MSDC-0602K, nitazoxanide).

There are other drugs (ASC-41, TERN-501, VK-2809) for this indication with a similar MOA to resmetirom (a thyroid hormone receptor beta agonist) in Phase II development at this time.

Rusfertide acetate

Polycythemia vera

There are no other drugs for this indication in Phase II development at this time.

Seladelpar

Primary biliary cholangitis (primary biliary cirrhosis)

  • Bile acid receptor agonist (ASC-42 , TQA-3526 , ZG-5266);
  • Catenin Beta 1 inhibitor (PRI-724);
  • Fibroblast growth factor receptor 1 agonist (aldafermin);
  • Gamma-Aminobutyric Acid Type A receptor subunit antagonist (golexanolone);
  • Ileal sodium/bile acid cotransporter (volixibat);
  • T-lymphocyte activation antigen CD80 inhibitor (rhudex).

There are no other drugs for this indication with the same MOA as seladelpar (a PPAR-delta agonist) in Phase II development at this time.

Setmelanotide

Obesity

  • Calcitonin receptor agonist (cagrilintide);
  • Enteropeptidase inhibitor (sco-792);
  • Gastric inhibitory polypeptide receptor agonist (ct-868, retatrutide);
  • Glucagon like peptide 1 receptor agonist (bi-456906, danuglipron, ecnoglutide, efinopegdutide, ly-3502970, pegapamodutide, pemvidutide);
  • Glucocorticoid receptor antagonist (miricorilant);
  • Combinations (acarbose + orlistat; leucine + metformin + sildenafil; leucine + sildenafil);
  • Other with an undisclosed MOA (bittera, cbl-514, mbl-949, nn-9775, nov-db2, novob, rzl-12).

There are no other drugs for this indication with the same MOA as setmelanotide (a melanocortin-4 receptor agonist) in Phase II development at this time.

Soticlestat

Lennox-Gastaut syndrome; Dravet syndrome (severe myoclonic epilepsy of infancy)

  •  5-hydroxytryptamine receptor 2C agonist (LP-352);
  • Metabotropic glutamate receptor 1 antagonist (JBPOS-0101).

There are no other drugs for this indication with the same MOA as soticlestat (a cholesterol 24-hydroxylase inhibitor) in Phase II development at this time.

Sotatercept

Pulmonary arterial hypertension (PAH)

  • ACE 2 replacement (APN-01);
  • Atrial natriuretic peptide receptor 1 agonist (ularitide);
  • Calcineurin inhibitor (tacrolimus);
  • Cgmp specific 3',5' cyclic phosphodiesterase inhibitor (vardenafil inhalation);
  • Dopamine beta hydroxylase inhibitor (zamicastat).histone deacetylase inhibitor (valproic acid);
  • Macrophage colony stimulating factor 1 receptor inhibitor (seralutinib);
  • Prostacyclin receptor agonist (epoprostenol inhalation, treprostinil palmitil inhalation);
  • Tryptophan 5 monooxygenase inhibitor (rodatristat).

There are no other drugs for this indication with the same MOA as sotatercept (an activin receptor type IIA-Fc fusion protein) in Phase II development at this time.

Spesolimab*

Generalized pustular psoriasis (GPP)

There are no other drugs for this indication in Phase II development at this time.

Tremelimumab

Hepatocellular carcinoma

  • Aldo keto reductase family 1 member C3 inhibitor (TH-3424);
  • Arginine depletor (BCT-100);
  • Aurora kinase A inhibitor (TT-00420);
  • Catenin beta 1 inhibitor (PRI-724);
  • C-C chemokine receptor type 2 antagonist (BMS-813160);
  • CCAAT/enhancer binding protein alpha activator (MTL-CEBPA);
  • Cell therapy (various, activated T lymphocytes; llixadencel, NRT-01);
  • Cyclic AMP dependent transcription factor ATF 5 inhibitor (ST-101);
  • Cyclin dependent kinase 1 inhibitor (milciclib);
  • Cyclin dependent kinase 2 inhibitor (fadraciclib);
  • Cytotoxic T lymphocyte protein 4 inhibitor (ADG-126, BMS-986249, ONC-392, PSB-205, zalifrelimab);
  • Cytotoxic to cells expressing:
  • CD276 Antigen (MGC-018);
  • Epidermal growth factor receptor (AFM-24I);
  • Fibronectin (dodekin);
  • Glypican 3 (codrituzumab);
  • Intercellular adhesion molecule 1 (gebasaxturev);
  • Stabilin 1 (bexmarilimab);
  • Trophoblast glycoprotein (naptumomab estafenatox);
  • Dickkopf related protein 1 inhibitor (DKN-01);
  • DNA polymerase inhibitor (fostroxacitabine bralpamide);
  • DNA synthesis inhibitor (cisplatin + vinblastine; evofosfamide, tirapazamine);
  • Dual specificity protein kinase TTK inhibitor (NMS-153);
  • Epidermal growth factor receptor antagonist (EMB-01, fmab-2);
  • F box like/WD repeat containing protein TBL1X inhibitor (tegavivint);
  • Fibroblast growth factor receptor 4 antagonist (roblitinib);
  • Gene-modified cell therapy (various, BOXR-1030, ET-1402, Fully Human B7H3 CAR-T Cells, IMA-203CD8, JWATM-204, LIOCYXM-004, SCG-101);
  • Growth/differentiation factor 15 inhibitor (visugromab);
  • Hepatocyte growth factor receptor inhibitor (TPX-0022);
  • Histone deacetylase inhibitor (tefinostat);
  • Inducible T cell costimulator agonist (alomfilimab);
  • Interferon beta activator (Voyager-V1);
  • Interleukin 2 receptor agonist (ANV-419, BNT-151, SAR-444245);
  • Interleukin 8 inhibitor (BMS-986253);
  • Interleukin 12 receptor agonist (BMS-986415);
  • Interleukin 15 receptor subunit alpha agonist (SOT-101);
  • Interleukin 18 receptor 1 agonist (ST-067);
  • Interleukin 27 receptor antagonist (SRF-388);
  • Leukocyte immunoglobulin like receptor subfamily B member 1 antagonist (SAR-444881);
  • Lymphocyte activation gene 3 protein inhibitor (INCAGN-2385);
  • MAP kinase interacting serine/threonine protein kinase 1 inhibitor (tomivosertib);
  • Mast/stem cell growth factor receptor Kit inhibitor (MG-010 + sorafenib);
  • Mitogen activated protein kinase kinase kinase kinase 1 inhibitor (CFI-402411, PRJ-13024);
  • Myc proto oncogene protein inhibitor (OTX-2002);
  • Na+/K+ exchanging atpase inhibitor (RX-108);
  • Nuclear factor Kappa B inhibitor (ACT-001);
  • Opioid receptor antagonist (metenkefalin);
  • Peroxisome proliferator activated receptor alpha antagonist (TPST-1120);
  • Phosphatidylinositol 4,5 bisphosphate 3 kinase catalytic subunit delta isoform inhibitor (BGB-10188);
  • Programmed cell death 1 ligand 1 inhibitor (HB-0036, INCB-86550, spartalizumab);
  • Protein cereblon activator (avadomide);
  • Protein tyrosine phosphatase Type IVA 3 inhibitor (PRL3-ZUMAB);
  • Receptor tyrosine protein kinase ERBB 3 antagonist (HMBD-001);
  • Serine/threonine protein kinase mtor inhibitor (sapanisertib);
  • Serine/threonine protein kinase receptor R3 inhibitor (ascrinvacumab);
  • Serine/threonine protein kinase PLK1 inhibitor (CYC-140);
  • Sialic acid binding Ig like lectin 15 inhibitor (NC-318);
  • Stimulator of interferon genes protein activator (CDK-002, IMSA-101, SB-11285);
  • Telomerase reverse transcriptase inhibitor (KML-001);
  • TGF beta receptor Type 1 inhibitor (galunisertib);
  • T lymphocyte activation antigen CD80 inhibitor (BA-3071);
  • Toll like receptor 9 agonist (SD-101);
  • Transferrin receptor protein 1 antagonist (CX-2029);
  • Vaccine (GNOSPV-02, IMA-970A, maveropepimut-s, ombipepimut-s, TAEK-VAC);
  • Other with an undisclosed MOA (ABX-196, ecubectedin, foslinanib, OC-001, phosphoethanolamine, PM-8003, SAR-444200).

There are other drugs (ADG-126, BMS-986249, ONC-392, PSB-205, zalifrelimab) for this indication with a similar MOA to tremelimumab (a cytotoxic T lymphocyte protein 4 inhibitor) in Phase II development at this time.

Ulotaront

Schizophrenia

  • Alpha 2 adrenergic receptor antagonist (TR-01);
  • D amino acid oxidase inhibitor (RSD-7);
  • Camp and camp inhibited cgmp 3',5' cyclic phosphodiesterase 10A inhibitor (MK-8189);
  • Cannabinoid receptor 1 agonist (cannabidiol);
  • D1A dopamine receptor antagonist (tetrahydropalamatine);
  • 5-hydroxytryptamine receptor 2 antagonist (iloperidone LAI);
  • Muscarinic acetylcholine receptor M4 agonist (emraclidine);
  • Potassium voltage gated channel subfamily C member 1 activator (AUT-00206);
  • Sodium and chloride dependent glycine transporter 1 inhibitor (bitopertin).

There is one other drug (ralmitaront) for this indication with a similar MOA to ulotaront (a TAAR1 agonist) in Phase II development at this time.

Zolbetuximab

 

Refer to information under “bemarituzumab”

Zoliflodacin

Uncomplicated cervical and urethral gonorrhea

There are no other drugs for this indication in Phase II development at this time.

*Under review by Health Canada.

Abbreviations: ACE: angiotensin converting enzyme; ASA: acetylsalicylic acid; BTK: Bruton's tyrosine kinase; CETP: cholesteryl ester transfer protein; CNS: central nervous system; JAK: Janus Kinase; MOA: mechanism of action; PPAR: peroxisomal proliferator-activated receptor; TAAR1: trace amine associated receptor 1.

Data source: GlobalData Healthcare database (accessed September 2022).

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  195. A Multicenter, Randomized, Active-controlled, Double-blind, Double-dummy, Parallel Group Clinical Trial, Investigating the Efficacy, Safety, and Tolerability of Continuous Subcutaneous ND0612 Infusion in Comparison to Oral IR-LD/CD in Subjects With Parkinson's Disease Experiencing Motor Fluctuations (BouNDless). NCT04006210 (recruiting). https://clinicaltrials.gov/ct2/show/NCT04006210?term=NCT04006210&draw=2&rank=1.
  196. An Open-label, Long-term Extension Study of Brazikumab in Participants With Moderately to Severely Active Crohn's Disease (INTREPID OLE). NCT03961815 (enrolling by invitation). https://clinicaltrials.gov/ct2/show/NCT03961815?term=NCT03961815&draw=2&rank=1.
  197. A 52-Week, Multicenter, Randomized, Double-blind, Placebo and Active-Controlled, Operationally Seamless Phase 2b/3, Parallel-group Study to Assess the Efficacy and Safety of Brazikumab in Participants With Moderately to Severely Active Crohn's Disease (INTREPID Lead-In). NCT03759288 (recruiting). https://clinicaltrials.gov/ct2/show/NCT03759288?term=NCT03759288&draw=2&rank=1.
  198. A Phase 3 Prospective, Randomized, Double-blinded, Placebo-controlled Clinical Study to Evaluate the Efficacy and Safety of RBX2660 (Microbiota Suspension) for the Prevention of Clostridium Difficile InfectionNCT03244644 (completed). https://clinicaltrials.gov/ct2/show/NCT03244644?term=NCT03244644&draw=2&rank=1
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  200. Ferring and Rebiotix Present Landmark Phase 3 Data Demonstrating Superior Efficacy of Investigational RBX2660 Versus Placebo to Reduce Recurrence of C. difficile Infection. May 21, 2021. https://www.rebiotix.com/efficacy-rbx2660-vs-placebo-reduce-recurrence-c-difficile-infection/
  201. A Phase 3 Open-Label Clinical Study to Evaluate the Safety and Tolerability of Rebiotix RBX2660 (Microbiota Suspension) in Subjects With Recurrent Clostridium Difficile Infection. NCT03931941 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT03931941?term=NCT03931941&draw=2&rank=1.
  202. A Phase III, Randomized, Multicenter, Parallel-Group, Double-Blind, Double-Dummy Study in Adolescent and Adult Female Participants Comparing the Efficacy and Safety of Gepotidacin to Nitrofurantoin in the Treatment of Uncomplicated Urinary Tract Infection (Acute Cystitis). NCT04020341 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT04020341?term=NCT04020341&draw=2&rank=1.
  203. A Phase III, Randomized, Multicenter, Parallel-Group, Double-Blind, Double-Dummy Study in Adolescent and Adult Female Participants Comparing the Efficacy and Safety of Gepotidacin to Nitrofurantoin in the Treatment of Uncomplicated Urinary Tract Infection (Acute Cystitis). NCT04187144 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT04187144?term=NCT04187144&draw=2&rank=1.
  204. EAGLE-2 and EAGLE-3 phase III trials for gepotidacin stopped early for efficacy following pre-planned interim analysis by Independent Data Monitoring Committee Issued: London UK. November 3, 2022. https://www.gsk.com/en-gb/media/press-releases/gsk-announces-phase-iii-trials-for-gepotidacin/
  205. New antibiotic appears to be effective against urinary tract infections, drug company says. November 3, 2022. https://www.cnn.com/2022/11/03/health/new-uti-antibiotic-gepotidacin/index.html
  206. GSK to submit drug application for new antibiotic to US FDA. November 4, 2022. https://www.pmlive.com/pharma_news/gsk_to_submit_drug_application_for_new_antibiotic_to_us_fda_1480238
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  208. PhaseBio Announces Successful Pre-BLA Meeting with U.S. FDA for Bentracimab. May 16, 2022. https://www.businesswire.com/news/home/20220516005342/en/PhaseBio-Announces-Successful-Pre-BLA-Meeting-with-U.S.-FDA-for-Bentracimab
  209. Danicopan (ALXN2040) add-on to Ultomiris or Soliris met primary endpoint in ALPHA Phase III trial for patients with paroxysmal nocturnal haemoglobinuria who experience clinically significant extravascular haemolysis. September 16, 2022. https://www.astrazeneca.com/media-centre/press-releases/2022/danicopan-phase-iii-trial-met-primary-endpoint.html
  210. A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH). NCT04469465 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT04469465?term=NCT04469465&draw=2&rank=1.
  211. Gene Therapy for Hemophilia Is on the Brink of FDA Approval. November 17, 2022. https://www.managedhealthcareexecutive.com/view/gene-therapy-for-hemophilia-is-on-the-brink-of-fda-approval
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  213. Press Release: Fitusiran prophylaxis reduced bleeds by 61% in people with hemophilia A or B, with or without inhibitors, compared to prior factor or bypassing agent prophylaxis. July 10, 2022. https://www.sanofi.com/en/media-room/press-releases/2022/2022-07-10-13-45-00-2476896
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  217. Tuberculosis Pipeline Landscape Analysis of 38+ Companies by DelveInsight. May 4, 2022. https://www.globenewswire.com/en/news-release/2022/05/04/2436001/0/en/Tuberculosis-Pipeline-Landscape-Analysis-of-38-Companies-by-DelveInsight.html
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  225. A Randomized, Multicenter, Parallel-group, Phase III Study to Compare the Efficacy of Arfolitixorin Versus Leucovorin in Combination With 5 Fluorouracil, Oxaliplatin, and Bevacizumab in Patients With Advanced Colorectal Cancer. NCT03750786 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT03750786?term=NCT03750786&draw=2&rank=1.
  226. A Phase 3 Multicenter, Randomized, Double-Blind, Placebo Controlled Study to Determine the Efficacy of Topical SGX301 (Synthetic Hypericin) and Fluorescent Bulb-Light Irradiation for the Treatment of Cutaneous T-Cell Lymphoma. NCT02448381 (completed). https://clinicaltrials.gov/ct2/show/NCT02448381?term=NCT02448381&draw=2&rank=1.
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  229. A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial Testing Ipatasertib Plus Abiraterone Plus Prednisone/Prednisolone, Relative to Placebo Plus Abiraterone Plus Prednisone/Prednisolone in Adult Male Patients With Asymptomatic or Mildly Symptomatic, Previously Untreated, Metastatic Castrate-Resistant Prostate Cancer. NCT03072238 (active, but not recruiting). https://clinicaltrials.gov/ct2/show/NCT03072238?term=NCT03072238&draw=2&rank=1
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