Meds Pipeline Monitor 2023

Contact Information

Patented Medicine Prices Review Board
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Acknowledgements

This report was prepared by the Patented Medicine Prices Review Board (PMPRB) as part of the National Prescription Drug Utilization Information System (NPDUIS) research 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 to Brian O’Shea and Kevin Pothier for their oversight in the development of the report. The PMPRB also wishes to acknowledge the editorial contributions of Shirin Paynter.

Disclaimer

The NPDUIS research initiative 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. (2024). Meds Pipeline Monitor, 2023. 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 2023

  • As of April 2024, the pipeline contained over 12,000 new medicines in various stages of clinical development, compared to just over 9,000 the year before. The number of drugs in the pipeline is increasing by an average of 19% per year since 2019.
  • Oncology continues to dominate the therapeutic mix in 2023, with cancer treatments representing one third (33%) of medicines in all phases of clinical trials. Treatments for infectious diseases and central nervous system diseases held the second and third largest share of the pipeline, at 13% and 12%, respectively.
  • On average, 20% of medicines in Phase III clinical trials and pre-registration in 2023 had an early orphan designation approved through the U.S. FDA or the EMA, which is roughly a 33% decrease from previous years.
  • Twenty new medicines were selected for the 2023 new medicines list (Table 4) based on their potential to impact the Canadian healthcare system. Fourteen of the medicines listed in this year’s report (Tables 4 through 7) have forecasted global annual revenues over US $1 billion by 2029. 
  • Of the 51 new and retained medicines listed in the previous edition (MPM 2022), 15 received market authorization, 23 were retained on this year’s list as they continued to satisfy the selection criteria, and 13 were removed as their clinical trials were discontinued or they no longer meet the selection criteria.
  • Six new medicines under review by Health Canada were selected for this report as they have a novel mechanism of action or have demonstrated improved efficacy and/or safety in clinical trials.

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.
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.
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

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 vii

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 (2019-2023), and a breakdown of the various therapeutic areas for each phase of clinical development.

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 2023 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 April 2024 and the selected medicine candidates for this year’s report have been validated as of August 30, 2024.

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 in earlier stages of the pipeline (i.e., Phase II). 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 Meds Pipeline Monitor 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 Türkiye).

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 Türkiye).

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 and cell 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 Türkiye). 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 Meds Pipeline Monitor

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 Meds Pipeline Monitor

Attribute Relevance Data sources
Phase III 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 U.S. 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.

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), U.S. 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 Türkiye). 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 U.S. 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 April 2024 and are validated as of the end of August 2024. 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 2023 Pipeline

The number of new pharmaceutical developments in the pipeline is increasing year over year. In 2023, over 12,000 new medicines were undergoing clinical evaluation, which has been increasing by an average of 19% per year since 2019.

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, 2019-2023 Figure 2. Number of pipeline medicines in each stage of clinical evaluation
Figure description

A bar graph shows the total number of new medicines in each phase of the pipeline by their highest phase of development from 2019 to 2023. Totals are given for each year and phase.

  Phase I Phase II Phase III Pre-registration

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

2023

5,319

4,979

1,671

234

Data source: GlobalData Healthcare database (accessed April 2024); 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 2023, cancer treatments dominated the therapeutic mix in each phase of the pipeline, accounting for one third (33%) of medicines in all phases of clinical evaluation. Other important pipeline therapies include those for infectious diseases (13%) and central nervous system therapies (12%).

Figure 3a. Therapeutic class distribution of pipeline medicines by phase of clinical evaluation, 2023 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

38%

33%

20%

24%

33%

Infectious disease

12%

13%

18%

8%

13%

Central Nervous System

12%

13%

12%

10%

12%

Metabolic disorders

6%

5%

8%

10%

6%

Immunology

6%

5%

5%

7%

5%

Gastrointestinal

5%

5%

3%

4%

5%

Cardiovascular

5%

5%

9%

6%

5%

Respiratory

4%

5%

4%

2%

4%

Musculoskeletal disorders

2%

3%

4%

4%

3%

Dermatology

2%

5%

4%

3%

4%

Ophthalmology

2%

3%

6%

4%

3%

Genetic disorders

1%

2%

1%

2%

1%

Hematological disorders

1%

2%

3%

5%

2%

Other

4%

2%

4%

10%

3%

Data source: GlobalData Healthcare database (accessed April 2024).

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 2023.

Figure 3b. Top indications for major therapeutic areas in the pipeline, 2023 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

33%

Solid Tumor (228)

Non-Small Cell Lung Cancer (94)

Gastric Cancer (49)

Head and Neck Squamous Cell Carcinoma (47)

Colorectal Cancer (46)

Triple-Negative Breast Cancer (37)

Ovarian Cancer (36)

Relapsed/Refractory Acute Myeloid Leukemia (35)

Pancreatic Cancer (33)

Diffuse Large B-Cell Lymphoma (33)

Melanoma (31)

Infectious disease

13%

COVID-19 and associated symptoms (29)

Human Immunodeficiency Virus (HIV) (14)

Acinetobacter Infections (6)

Influenza A and B Virus Infections (6)

Respiratory Syncytial Virus (RSV) Infections (5)

Central Nervous System

12%

Alzheimer’s Disease (29)

Parkinson’s Disease (22)

Post-Operative Pain (15)

Amyotrophic Lateral Sclerosis (15)

Neuropathic Pain (13)

Multiple Sclerosis (9)

Opium Addiction (9)

Epilepsy (8)

Autism Spectrum Disorder (5)

Metabolic disorders

6%

Obesity (15)

Type 2 Diabetes (12)

Type 1 Diabetes (7)

Dyslipidemia (7)

Hypercholesterolemia (5)

Immunology

5%

Rheumatoid Arthritis (15)

Autoimmune Disorders (12)

Systemic Lupus Erythematosus (11)

Plaque Psoriasis (7)

Systemic Sclerosis (6)

Gastrointestinal

5%

MASH (20)

Ulcerative Colitis (18)

Crohn’s Disease (8)

Inflammatory Bowel Disease (5)

Irritable Bowel Syndrome (5)

Liver Fibrosis/Cirrhosis (3)

Cardiovascular

5%

Heart Failure (9)

Pulmonary Arterial Hypertension (8)

Hypertension (3)

Thrombosis (3)

Cardiomyopathy (3)

Respiratory

4%

Idiopathic Pulmonary Fibrosis (14)

Cystic Fibrosis (4)

Streptococcal Pneumonia (4)

Pulmonary Fibrosis (3)

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 2023 pipeline. Figure 4 provides the shares of orphan designated medicines for all phases in the pipeline from 2020-2023. Orphan designated medicines make up a greater share in the later stages of the pipeline, increasing from 6% in Phase I to 22% in pre-registration in 2023.

Figure 4. Share of orphan medicines in the pipeline by highest phase of clinical evaluation, 2020-2023 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 2023.

  Phase of Development 2020 2021 2022 2023

Orphan designation

Phase I

8%

7%

7%

6%

Phase II

20%

21%

22%

19%

Phase III

33%

36%

31%

18%

Pre-registration

40%

30%

31%

22%

No orphan designation

Phase I

92%

93%

93%

94%

Phase II

80%

79%

78%

81%

Phase III

67%

64%

69%

82%

Pre-registration

60%

70%

69%

78%

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 Türkiye). 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 April 2024).

Meds Pipeline Monitor 2023

The following tables include selected new medicine candidates for 2023 (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 1,905 pipeline medicines in Phase III and pre-registration in 2023, twenty (20) 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 investigation (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 2023 new medicines list. The description for each new medicine listed in the 2023 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 A2) 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 28.9% of developmental candidates advancing to Phase III.1

Of the new medicines featured in previous reports, 23 were retained as recent evidence continues to support promising clinical benefit and satisfies the selection criteria (Table 5). Fifteen of the 2022 pipeline medicines have received market authorization in the US, Europe, or Canada as of August 30, 2024 (Table 6), while 13 were removed from the list as their clinical trials were discontinued or they no longer fulfill the selection criteria.

Screening biosimilars

The availability of biosimilars could significantly impact costs in a wide range of therapeutic areas. 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 2023

Cardiovascular

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

Aficamten

Cytokinetics Inc.

  • Increased safety and efficacy
  • Breakthrough

Hypertrophic cardiomyopathy

  • Clinical trials in Canada
  • Rare or orphan designation
  • It is a selective cardiac myosin inhibitor that reduces left ventricular outflow tract gradients by mitigating cardiac hypercontractility.
  • Administered orally.

Clinical trials

  • It has similar efficacy to mavacamten (Camzyos), also a cardiac myosin inhibitor, but it has a shorter half-life (t1/2) and fewer drug-drug interactions,2 suggesting improved safety. The shorter half-life allows for the dose to be uptitrated quickly, resulting in earlier symptomatic relief.3
  • One Phase III trial has been completed;4 others are ongoing.5, 6,7,8
  • No other drug for this indication was identified in Phase II development at this time.9

Forecasted revenue

  • Total global annual revenue forecasted to be $2.3 billion by 2029.*

Nerinetide

NoNO Inc.

  • Increased safety and efficacy

Acute ischemic stroke

  • Clinical trials in Canada
  • Rare or orphan designation
  • It acts as a neuroprotective eicosapeptide by inhibiting the post-synaptic density protein 95 (PSD-95). 
  • Administered as an intravenous infusion.

Clinical trials

  • There is promising evidence for improving the outcomes of patients with acute ischemic stroke.10
  • The Phase III trials have been completed.11,12,13
  • There are other drugs in Phase II development (n=10), but none with the same mechanism of action as nerinetide.14  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Pelacarsen sodium

Novartis AG

  • Increased safety and efficacy
  • Fast Track

Cardiovascular disease; Hyperlipidemia

  • Clinical trials in Canada
  • It is a first in class ligand-conjugated antisense (LICA) medicine designed to inhibit the production of apolipoprotein(a), or apo(a), in the liver to offer a direct approach for reducing Lp(a). Elevated Lp(a) is recognized as an independent, genetic cause of cardiovascular disease (CVD). It inhibits elevated levels of apolipoprotein(Lp)(a). There are no approved drug therapies that are designed to target Lp(a) with the goal of lowering the Lp(a) level in patients who have increased risk.
  • Administered as a monthly subcutaneous injection.

Clinical trials

  • Evidence to date shows that a single dose of pelacarsen yields optimal results with persisting substantial reductions in Lp(a) levels, potentially enhancing CVD risk reduction.15
  • There are ongoing Phase III trials.16,17,18,19
  • There are other drugs in Phase II development (n=5), but none with the same mechanism of action as pelacarsen.  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $625 million by 2029.*

Central Nervous System

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

Fosigotifator (ABBVCLS-7262)

Calico Life Sciences LLC

  • Novel mechanism

Amyotrophic lateral sclerosis (ALS)

  • Clinical trials in Canada
  • It is a eukaryotic translation initiation factor 2 subunit beta (EIF2B) activator. 
  • Administered orally once a day.

Clinical trials

  • In Phase II trials, ABBV-CLS-7262 increased eIF2B activity and inhibited the Integrated Stress Response (ISR) in blood cells collected from trial participants.20 The ISR is activated in people with ALS causing a reduction in normal protein synthesis, an increase in production of stress proteins, and formation of stress granules containing TDP-43.
  • There is an ongoing Phase II/III trial (enrolling by invitation)21; 300 patients to participate.
  • There are other drugs in Phase II development (n>10), but none with the same mechanism of action as ABBVCLS-7262.22 See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Iclepertin

Boehringer Ingelheim International GmbH

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Cognitive impairment associated with schizophrenia (CIAS)

  • Clinical trials in Canada
  • It is a glycine transporter 1 (GlyT1) inhibitor.
  • Administered orally.

Clinical trials

  • A Phase II study has demonstrated that iclepertin is safe and well tolerated in patients with schizophrenia and improves cognition.23
  • It has the potential to become the first treatment option for cognitive impairment associated with schizophrenia.24
  • Phase III trials are ongoing.25,26,27,28, 29,30
  • There are other drugs in Phase II development (n=5) but none with the same mechanism of action as iclepertin.31  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Resiniferatoxin

Grunenthal GmbH

  • Increased safety and efficacy
  • Breakthrough

Osteoarthritis pain

  • Clinical trials in Canada
  • It is an ultrapotent capsaicin analog that acts as a transient receptor potential vanilloid subtype 1 (TRPV1) agonist. Its administration can reversibly defunctionalise TRPV1-expressing nociceptors. This may result in long-lasting pain relief.
  • Administered by intraarticular injection.

Clinical trials

  • The therapeutic window of resiniferatoxin is broad, allowing for the full desensitization of pain perception and neurogenic inflammation without causing unacceptable side effects.32
  • If approved, it has the potential to become a meaningful non-opioid treatment option providing long-lasting pain relief and functional improvement of the affected joint, combined with a favourable safety profile.33
  • Phase III trials are undergoing a protocol change.
  • There are other drugs in Phase II development (n>10) with one having the same mechanism of action as resiniferatoxin.34  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $24 million by 2029.*

Xanomeline-trospium (KarXT)

Karuna Therapeutics Inc.

  • Increased safety and efficacy

Schizophrenia; Psychosis

  • Clinical trials in Canada
  • It is a dual M1/M4 muscarinic acetylcholine receptor agonist that does not block D2 dopamine receptors. KarXT combines xanomeline with the peripherally restricted muscarinic receptor antagonist trospium chloride with the goal of ameliorating xanomeline-related adverse events associated with peripheral muscarinic receptors.
  • Administered orally.

Clinical trials

  • In the EMERGENT-2 Phase III trial, KarXT was effective in reducing positive and negative symptoms in schizophrenia and was generally well tolerated.35 A meaningful response was defined as a 30% or greater reduction in total PANSS score by week 5. Using this definition, 55% of persons on KarXT responded versus 28% on placebo. This difference was both statistically significant and clinically meaningful.36
  • Some Phase III trials for schizophrenia have been completed,37,38,39 others are ongoing.40,41,42,43  One was terminated (company’s business decision).44 There are ongoing Phase III trials for psychosis associated with Alzheimer’s disease.45,46,47
  • It is currently under review by the U.S. FDA.
  • There are other drugs in Phase II development (n=4) with several having the same mechanism of action as xanomeline-trospium.48  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $3.3 billion by 2029.*

Dermatology

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

Prademagene zamikeracel

Abeona Therapeutics Inc.

  • Increased safety and efficacy
  • Gene or cell therapy

Epidermolysis bullosa

  • Biologic medicine
  • Rare or orphan designation
  • It is designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome. 49
  • Applied topically, as engineered autologous epidermal sheets.

Clinical trials

  • Findings from the Phase III trial50 showed 81.4% of prademagene-treated wounds achieved 50% or greater wound healing compared with 16.3% of untreated control wounds (p<.001). Additionally, a statistically significant improvement in pain reduction associated with wound dressing changes was observed with the prademagene when compared with untreated control wounds (p=.0002).51,52
  • Another Phase III trial is ongoing.53
  • Although FDA issued a complete response letter in April 2024, specifying chemistry, manufacturing and controls issues that must be addressed, the company has since met with the FDA and is planning to resubmit.54
  • There are other drugs in Phase II development (n=4) with one having the same mechanism of action as prademagene zamikeracel. 55  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Gastrointestinal Disorders

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

Efruxifermin

Akero Therapeutics Inc.

  • Novel mechanism
  • Breakthrough
  • Fast Track

 

Metabolic dysfunction-associated steatohepatitis (MASH)

  • Clinical trials in Canada
  • It is a bivalent, long-acting fibroblast growth factor 21 (FGF21) analog that is designed to reduce liver fat and inflammation, reverse fibrosis, increase insulin sensitivity and improve lipids.
  • Administered subcutaneously.

Clinical trials

  • In Phase II studies, treatment with efruxifermin significantly reduced hepatic fat fraction (HFF) in patients with F1-F3 stage NASH (now referred to as MASH), with an acceptable safety profile.56,57
  • When added to glucagon-like peptide-1 receptor agonists (GLP-1RAs), its tolerability appeared comparable to that of either drug alone, while also significantly reducing HFF and noninvasive markers of fibrosis in patients with MASH and type 2 diabetes (T2D). Liver health in patients already on a GLP-1RA may be further improved by addition of efruxifermin.58
  • Phase III trials are ongoing.59,60
  • There are other drugs in Phase II development (n>10) with one having the same mechanism of action as efruxifermin.61  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $546 million by 2029.*

Obefazimod

Abivax SA

  • Novel mechanism

Ulcerative colitis

  • Clinical trials in Canada
  • It is a small molecule with anti-inflammatory properties through the specific and selective upregulation of miR-124 expression.
  • Administered orally.

Clinical trials

  • Findings from the maintenance phases of Phase II trials showed that long-term obefazimod treatment provides continued improvement in clinical symptoms of disease, with a substantial proportion of patients in clinical remission, and an overall good safety profile.62
  • Phase III trials are ongoing.63,64,65
  • There are other drugs in Phase II development (n>10), but none with the same mechanism of action as obefazimod.66  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $715 million by 2029.*

Genetic disorders

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

Fazirsiran sodium

Arrowhead Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Breakthrough
  • Fast Track
  • Gene or cell therapy

Alpha-1 antitrypsin deficiency (A1AD)

  • Clinical trials in Canada
  • Biologic medicine
  • Rare or orphan designation
  • It is a small, interfering RNA (siRNA) that inhibits the mutant alpha-1 antitrypsin (Z-AAT) protein.
  • Administered subcutaneously.

Clinical trials

  • In a Phase II trial, fazirsiran was associated with a strong reduction of Z-AAT (the Z allele of alpha1-antitrypsin protein) concentrations in the serum and liver and concurrent improvements in liver enzyme concentrations.
  • Phase III trials are ongoing.67,68,69
  • There are other drugs in Phase II development (n=3) with two having the same mechanism of action as fazirsiran.  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $495 million by 2029.*

Genito Urinary System And Sex Hormones

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

Inaxaplin (VX19-147)

Vertex Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Breakthrough

Focal segmental glomerulosclerosis (FSGS); Chronic kidney disease (chronic renal failure)

  • Clinical trials in Canada
  • Rare or orphan designation
  • It is an inhibitor of Apolipoprotein L1 (APOL1) channel activity that reduces proteinuria in patients with APOL1-mediated kidney disease (AMKD).
  • Administered orally.

Clinical trials

  • In a Phase II trial, inaxaplin reduced proteinuria in participants with two APOL1 variants and focal segmental glomerulosclerosis.70  If this is confirmed in Phase III trials, it would a major advance in the therapy of proteinuric CKD.71
  • A Phase III trial is ongoing.72
  • There are other drugs in Phase II development (n=2), but none with the same mechanism of action as inaxaplin.73  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $539 million by 2029.*

Metabolic disorders

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

RGX-121

RegenxBio Inc.

  • Increased safety and efficacy
  • Fast Track
  • Gene or cell therapy

Mucopolysaccharidosis II (MPS II) (Hunter syndrome)

  • Clinical trials in Canada
  • Biologic medicine
  • Rare or orphan designation
  • It is a gene therapy designed to deliver a functional copy of the iduronate-2-sulfatase gene, using the NAV® AAV9 vector, to cells in the central nervous system (CNS).
  • Administered directly to the CNS using intracisternal or intracerebroventricular delivery, as a one-time dose.

Clinical trials

  • Interim data from the CAMPSIITE® trial supports that RGX-121 changes the course of disease by restoring the gene missing in boys with Hunter syndrome and has the potential to significantly improve vital brain function for patients living with this debilitating disease.74
  • The CAMPSIITE® trial is ongoing.75
  • There are other drugs in Phase II development (n=5) with one having the same mechanism of action as RGX-121.76  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $406 million by 2029.*

Oncology

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

Datopotamab deruxtecan

Daiichi Sankyo Co Ltd.

  • Increased safety and efficacy

Breast cancer, both HR-positive/HER2-negative and triple-negative

  • Clinical trials in Canada
  • Biologic medicine
  • It is a TROP2-targeted antibody-drug conjugate. TROP2 is a transmembrane protein sporadically expressed in healthy tissue, but broadly expressed and associated with poor prognosis in HR-positive/HER2-negative and triple negative breast cancer.77

Clinical trials

  • In a Phase III trial, it demonstrated a statistically significant and clinically meaningful improvement for the dual primary endpoint of progression-free survival compared to investigator’s choice of chemotherapy in patients with unresectable or metastatic HR-positive, HER2-negative breast cancer previously treated with endocrine-based therapy and at least one systemic therapy. For the dual primary endpoint of overall survival (OS), interim results numerically favoured datopotamab deruxtecan over chemotherapy but were not mature at the time of data cut-off. The trial is ongoing and OS will be assessed at future analyses. 78
  • If approved, datopotamab deruxtecan has the potential to provide patients an efficacious and better tolerated alternative to conventional chemotherapy.79
  • The selective payload delivery of datopotamab deruxtecan, which is enabled by the selectively cleavable plasma-stable linker that releases deruxtecan after proteolytic processing by tumour cell–enriched lysosomal enzymes, reduces systemic exposure while achieving a sustained response, resulting in an improved benefit-risk profile. This may account for the comparatively low incidences of neutropenia and diarrhea in this study compared with sacituzumab govitecan, another TROP2-targeted antibody-drug conjugate marketed, as Trodelvy, in Canada since 2021.80
  • Phase III trials in breast cancer are ongoing.81,82,83,84,85
  • Under review by the U.S. FDA.86
  • While there are other drugs for breast cancer in Phase II development, there are no other drugs for all types of breast cancer targeted by datopotamab.87

Forecasted revenue

  • Total global annual revenue forecasted to be $4.4 billion by 2029.*

Gemcitabine (GemRIS)

Johnson & Johnson

  • Increased safety and efficacy
  • Breakthrough
  • Fast Track

Non-muscle invasive bladder cancer (NMIBC) (superficial bladder cancer); Muscle invasive bladder cancer (MIBC)

  • Clinical trials in Canada
  • Potential evergreening
  • It acts as a ribonucleoside diphosphate reductase subunit M1 inhibitor. It is a drug-device combination product designed to deliver a targeted, sustained release of gemcitabine into the bladder for weeks at a time.
  • Administered intravesically. It is installed in a physician’s office setting during a 3- to 5-minute procedure with no anesthesia.

Clinical trials

  • In a Phase II study, treatment with gemcitabine produced complete responses (CR) and was well tolerated in patients with Bacillus Calmette-Guérin–unresponsive, high-risk non–muscle-invasive bladder cancer (NMIBC).88  Additional data from the study show rapid achievement of CR with 98% achieving a CR within 12 weeks.89
  • Phase III trials are ongoing.90,91,92
  • There are other drugs in Phase II development (n=1), but none with the same mechanism of action as Gemcitabine.93  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $1 billion by 2029.*

Patidegib hydrochloride

Sol-Gel Technologies Ltd.

  • Increased safety and efficacy
  • Breakthrough

Gorlin syndrome (basal cell nevus syndrome/nevoid basal cell carcinoma syndrome)

  • Clinical trials in Canada
  • Rare or orphan designation
  • It is a hedgehog signaling pathway blocker.
  • Applied topically, as a gel.

Clinical trials

  • Patidegib is able to decrease tumour burden, reducing the adverse effects induced by systemic targeted therapies.94
  • A Phase III trial has been completed95 and one is ongoing.96 One trial was terminated early due to low blinded event rate; termination was not related to safety of the drug.97
  • There are other drugs in Phase II development (n=1), but none with the same mechanism of action as patidegib.98  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

Revumenib citrate (SNDX-5613)

Syndax Pharmaceuticals Inc.

  • Novel mechanism
  • Breakthrough
  • Fast Track
  • Priority Review

Acute lymphocytic leukemia (ALL); Acute lympho-blastic leukemia;

Refractory acute myeloid leukemia;

Relapsed acute myeloid leukemia

  • Clinical trials in Canada Rare or orphan designation
  • Rare or orphan designation
  • It is a selective, menin-mixed lineage leukemia inhibitor.
  • Administered orally.

Clinical trials

  • The breakthrough therapy designation (by U.S. FDA) underscores its potential as a first- and best-in-class therapy to meaningfully change the treatment paradigm for patients with R/R KMT2Ar acute leukemia, whether it presents clinically as acute myeloid leukemia or acute lymphocytic leukemia, in adults or children.99
  • Phase III trials have not been listed in clinicaltrials.gov. 
  • It is under review by the U.S. FDA.
  • There are other drugs in Phase II development (n=8) with several having the same mechanism of action as revumenib.100  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $583 million by 2029.*

Vorasidenib citrate

Les Laboratoires Servier SAS

  • Novel mechanism

Astrocytoma; Low-grade glioma; Oligodendroglioma

  • Clinical trials in Canada
  • Rare or orphan designation
  • It inhibits the activity of isocitrate dehydrogenase 1 (IDH1) and isocitrate dehydrogenase 2 (IDH2).
  • Administered orally.

Clinical trials

  • In the Phase III INDIGO trial, it significantly improved progression-free survival and delayed the time to the next intervention such as surgery, radiation, or chemotherapy, with a manageable safety profile.101 At 2 years follow-up, over 83% per cent of patients on vorasidenib did not require further treatment compared to only 27% on the placebo medication.
  • It is under review by the U.S. FDA and the EMA. If the Phase III study confirms these results, “relacorilant plus nab-paclitaxel has the potential to become a new standard of care.”102
  • The Phase III trial is ongoing.103
  • No other drug for all indications was identified in Phase II development at this time.104

Forecasted revenue

  • Forecasted annual global revenue unknown.

Respiratory

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

AD-109 (atomoxetine + R-oxybutynin)

Apnimed, Inc.

  • Increased safety and efficacy

Obstructive sleep apnea (OSA)

  • Clinical trials in Canada
  • It is a combination of R-oxybutynin, a selective antimuscarinic, and atomoxetine, a selective norepinephrine reuptake inhibitor. 
  • It has the potential to be the first oral pharmacologic that could both treat the underlying night-time airway obstruction and hypoxia that characterize OSA, as well as improve the daytime consequences of OSA, such as fatigue.
  • Administered orally, once daily at bedtime.

Clinical trials

  • Phase II studies showed that AD109, at the doses tested, achieved a statistically significant reduction of the Apnea-Hypopnea Index (AHI4, 4% desaturation definition for hypopneas) compared to placebo (p<0.001 vs. placebo). Dosing with AD109 led to clinically important reductions in AHI in most patients with mild, moderate and severe OSA.105,106,107
  • Currently, fewer than half of the people using PAP therapy are compliant long-term, leaving many people at risk from the consequences of untreated OSA, including a higher risk for stroke and heart attack.108
  • Phase III trials are ongoing.109,110
  • No other drug for this indication was identified in Phase II development at this time. 111

Forecasted revenue

  • Forecasted annual global revenue unknown.

Brensocatib

Insmed Inc.

  • Increased safety and efficacy
  • Breakthrough

Bronchiectasis

  • Clinical trials in Canada
  • It is a selective, reversible dipeptidyl peptidase 1 (cathepsin C) inhibitor that exhibits anti-inflammatory action through suppressing the activity of neutrophil serine proteases.
  • Administered orally.

Clinical trials

  • In a Phase II trial in patients with bronchiectasis, Brensocatib prolonged the time to the first exacerbation and led to fewer exacerbations than placebo.112,113
  • It is under review by the U.S. FDA.
  • A Phase III trial in bronchiectasis is ongoing.114
  • A Phase III trial in COVID-19 has been completed.115
  • There are other drugs in Phase II development (n=3), but none with the same mechanism of action as brensocatib.116  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $1.3 billion by 2029.*

* Consensus forecasts for global revenue data were collected from GlobalData, Q2-2024, and are given in US dollars.
Data source: GlobalData Healthcare database.

Table 5. Update on pipeline medicines retained from the 2022 Meds Pipeline Monitor

Cardiovascular

Medicine (Trade name) Company Indication(s) Update

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

Clinical trials

  • According to a cost-effectiveness study, abelacimab could offer a potential cost-savings of $50,000 USD and improvements of 1.5 quality-adjusted life years (QALY’s) per person over a lifetime horizon as compared to rivaroxaban, a direct oral anticoagulant.117
  • Two Phase III trials in cancer-associated thrombosis are ongoing; targeted to be completed in September 2025.118,119 
  • A Phase III trial in high-risk atrial fibrillation is ongoing; targeted to be completed in March 2025.120

Forecasted revenue

  • Forecasted annual global revenue unknown.

Etripamil

Milestone Pharmaceuticals Inc.

  • Novel mechanism

Supraventricular tachycardia

  • Clinical trials in Canada

Clinical trials

  • Positive results from the phase 3 RAPID clinical trial of etripamil nasal spray in patients with PSVT have been published.121
  • Two Phase III trials have been completed122,123  and others are still ongoing,124 including an open extension study by invitation.125
  • It has been submitted to the U.S. FDA for review.126

Forecasted revenue

  • Total global annual revenue forecasted to be $237 million by 2029.*

Obicetrapib

NewAmsterdam Pharma Company

  • Novel mechanism

Dyslipidemia; Heterozygous familial hypercholesterolemia (HeFH); Atherosclerosis

  • Clinical trials in Canada
  • Add-on Therapy

Clinical trials

  • According to a meta-analysis of trials, cholesteryl-ester transfer-protein inhibitors—including obicetrapib—are associated with reduced cardiovascular disease-related mortality and myocardial infarction.127
  • Phase III trials are still ongoing.128,129,130,131,132

Forecasted revenue

  • Total global annual revenue forecasted to be $582 million by 2029.*

Central Nervous System

Medicine (Trade name) Company Indication(s) Update

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

Clinical trials

  • Two Phase III trials have been completed133,134 and others are still ongoing.135,136

Forecasted revenue

  • Total global annual revenue forecasted to be $204 million by 2029.*

Latozinemab

(previously AL-001)

Alector Inc.

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

   

Frontotemporal dementia (FTD)

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

Clinical trials

  • In a Phase II study, latozinemab treatment for frontotemporal dementia showed no significant impact on disease progression, although the treatment was generally safe and well-tolerated.137
  • The Phase III trial is still ongoing; targeted to be completed in October 2027.138 A Phase III continuation study has been initiated.139
  • The U.S. FDA has granted breakthrough designation.140

Forecasted revenue

  • Total global annual revenue forecasted to be $9 million by 2029.*

Valiltramiprosate

(previously ALZ-801)

Alzheon Inc.

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

   

Alzheimer's disease (AD)

Clinical trials

  • The Phase III trial completed in July 2024.141
  • A long-term extension study has been added.142

Forecasted revenue

  • Forecasted annual global revenue unknown.

Midomafetamine (MDMA)

Lykos Therapeutics (formerly Multidisciplinary Association for Psychedelic Studies Public Benefit Corporation)

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Post-traumatic stress disorder (PTSD)

  • Clinical trials in Canada

Clinical trials

  • Another Phase III trial has been completed.143  Two Phase III trials have been initiated in 2024.144,145 
  • Phase III trials to date suggest that MDMA is superior to antidepressant medications for treating PTSD.146
  • Lykos Therapeutics has submitted an NDA (priority review) to the U.S. FDA.147  The FDA is convening a meeting of the Psychopharmacologic Drugs Advisory Committee on June 4, 2024, to review data.148

Forecasted revenue

  • Forecasted annual global revenue unknown.

ND-0612

(levodopa/carbidopa for subcutaneous infusion)

Neuroderm, a Mitsubishi Tanabe Pharma Corp subsidiary

  • Increased safety and efficacy

 

Parkinson's disease (PD)

  • Add-on Therapy

Clinical trials

  • Positive results from the Phase III trial have been reported149 and published.150
  • The Phase III trial is ongoing (has been extended to collect long-term safety data); targeted to be completed in February 2027.151

Forecasted revenue

  • Forecasted annual global revenue unknown.

Gastrointestinal disorders

Medicine (Trade name) Company Indication(s) Update

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

Clinical trials

  • Phase III results have been published. The percentage of patients who had a biochemical response and alkaline phosphatase normalization was significantly greater with seladelpar than with placebo. It also significantly reduced pruritus among patients who had moderate-to-severe pruritus at baseline.152
  • Two Phase III trials were completed153,154 and another is ongoing.155
  • The U.S. FDA accepted an NDA (priority review) in February 2024; a decision is expected by August 2024.156

Forecasted revenue

  • Total global annual revenue forecasted to be $478 million by 2029.*

Genetic disorders

Medicine (Trade name) Company Indication(s) Update

REC-2282

Recursion Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Fast Track

 

Neurofibromatosis type II (NF2)

  • Rare or orphan designation

Clinical trials

  • A Phase II/III trial is ongoing.157

Forecasted revenue

  • Total global annual revenue forecasted to be $163 million by 2029.*

Genito urinary system and sex hormones

Medicine (Trade name) Company Indication(s) Update

Gepotidacin mesylate

GlaxoSmithKline plc

  • Increased safety and efficacy
  • Novel mechanism

Cystitis; Urinary tract infections (UTI)

Clinical trials

  • It had been reported that the company expected to submit regulatory filings to the U.S. FDA in the first half of 2023.158  It now expects to file in the second half of 2024.159

Forecasted revenue

  • Total global annual revenue forecasted to be $434 million by 2029.*

Hematological disorders

Medicine (Trade name) Company Indication(s) Update

Bentracimab

SFJ Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Bleeding and clotting disorders

  • Biologic medicine

Clinical trials

  • The company filed a BLA in August 2024. The FDA has granted a priority review.160

Forecasted revenue

  • Forecasted annual global revenue unknown.

Fitusiran

Sanofi

  • Increased safety and efficacy
  • Fast Track

Hemophilia A;

Hemophilia B

  • Clinical trials in Canada
  • Rare or orphan designation

Clinical trials

  • Positive results from Phase III trials, that were previously reported in press releases, have now been published.161,162
  • The Phase III long-term study is ongoing, until November 2026.163
  • Another Phase III trial has been completed; no information on results is available at this time.164
  • A Phase III trial in pediatric patients is ongoing.165

Forecasted revenue

  • Total global annual revenue forecasted to be $459 million by 2029.*

Hormonal disorders

Medicine (Trade name) Company Indication(s) Update

Palopegteriparatide

Ascendis Pharma AS

  • Increased safety and efficacy

 

Hypoparathyroidism

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

Clinical trials

  • The U.S. FDA review time has been extended until August 2024.166

Forecasted revenue

  • Total global annual revenue forecasted to be $1.8 billion by 2029.*

Immunological disorders

Medicine (Trade name) Company Indication(s) Update

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

Clinical trials

  • Results of the Phase III trial have been published. Results show that monthly garadacimab administration significantly reduced hereditary angioedema attacks in patients aged 12 years and older compared with placebo and had a favourable safety profile.167
  • One Phase III trial was completed168 and another one is ongoing.169
  • The U.S. FDA accepted a BLA in December 2023; so has the EMA.170
  • Under review by Health Canada.171

Forecasted revenue

  • Forecasted annual global revenue unknown.

Infectious diseases

Medicine (Trade name) Company Indication(s) Update

Zoliflodacin

Innoviva Inc.

  • Increased safety and efficacy
  • Novel mechanism

 

Uncomplicated cervical and urethral gonorrhea

Clinical trials

  • Results of the Phase III trial have been positive.172

Forecasted revenue

  • Forecasted annual global revenue unknown.

Metabolic Disorders

Medicine (Trade name) Company Indication(s) Update

Birtamimab

Prothena Corp plc

  • Increased safety and efficacy
  • Fast Track

Primary systemic amyloidosis

  • Rare or orphan designation
  • Add-on Therapy

Clinical trials

  • Post hoc analyses from the Phase 3 VITAL trial has suggested that birtamimab plus standard of care confers a survival benefit in patients with advanced (Mayo Stage IV) AL amyloidosis.173
  • The Phase III confirmatory trial (AFFIRM-AL) is ongoing; targeted to be completed in June 2025.174

Forecasted revenue

  • Total global annual revenue forecasted to be $283 million by 2029.*

Oncology

Medicine (Trade name) Company Indication(s) Update

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

Clinical trials

  • Final analysis of Phase II trial showed that the combination of bemarituzumab-mFOLFOX6  led to numerically longer median progression-free survival  and overall survival compared with mFOLFOX6 alone.175
  • Phase III trials are ongoing.176,177

Forecasted revenue

  • Total annual global revenue forecasted to be $559 million by 2029.*

Navitoclax dihydrochloride

AbbVie Inc.

  • Increased safety and efficacy
  • Novel mechanism

Myelofibrosis

  • Clinical trials in Canada

Clinical trials

  • Phase III trials are ongoing.178,179 

Forecasted revenue

  • Total annual global revenue forecasted to be $496 million by 2029.*

Rusfertide acetate

Protagonist Therapeutics Inc.

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough

Polycythemia vera (PV)

  • Clinical trials in Canada

Clinical trials

  • Results from the Phase II trial have been published. Rusfertide treatment was associated with a mean hematocrit of less than 45% during the 28-week dose-finding period, and the percentage of patients with a response during the 12-week randomized withdrawal period was greater with rusfertide than with placebo.180
  • A Phase III trial is ongoing.181

Forecasted revenue

  • Forecasted annual global revenue unknown.

SGX-301 Hypericin sodium

(synthetic hypericin)

Soligenix Inc.

  • Increased safety and efficacy
  • Fast Track

Cutaneous T-cell lymphoma (CTCL)

  • Rare or orphan designation

Clinical trials

  • The company filed an NDA in December 2022 with the U.S. FDA. Upon preliminary review, the FDA determined that the NDA was not sufficiently complete to permit substantive review.182  After discussion with the FDA, a confirmatory Phase III trial is being undertaken.183,184

Forecasted revenue

  • Forecasted annual global revenue unknown.

Zolbetuximab

Astellas Pharma Inc.

  • Increased safety and efficacy
  • Novel mechanism

Adenocarcinoma of the gastroesophageal junction; Gastric cancer

  • Clinical trials in Canada
  • Add-on Therapy

Clinical trials

  • Approved in Japan (Vyloy; March 26, 2024).185
  • FDA reviewed (priority review) but did not approve due to insufficiencies pertaining to a pre-license inspection at a third-party manufacturing site for the agent.186

Forecasted revenue

  • Total annual global revenue forecasted to be $716 million by 2029.*

Ophthalmology

Medicine (Trade name) Company Indication(s) Update

Lenadogene nolparvovec

GenSight Biologics SA

  • Novel mechanism
  • Gene or cell therapy

Leber’s hereditary optic neuropathy (Leber optic atrophy)

  • Biologic medicine

Clinical trials

  • The efficacy of lenadogene nolparvovec in improving visual acuity in MT-ND4 Leber hereditary optic neuropathy (LHON) was confirmed in a large cohort of patients, compared to the spontaneous natural history decline.187
  • Phase III trials have been completed188,189,190 and one is ongoing.191

Forecasted revenue

  • Forecasted annual global revenue unknown.

* Consensus forecasts for global revenue data were collected from GlobalData, Q2-2024, and are given in US dollars.
Data source: GlobalData Healthcare database.

Table 6. Pipeline medicines from the 2022 Meds Pipeline Monitor that have gained market authorization

Cardiovascular

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Aprocitentan

(Tryvio)

Idorsia Pharmaceutical Ltd.

  • Increased safety and efficacy
  • Novel mechanism

Resistant hypertension

  • Clinical trials in Canada
  • Potential evergreening

Approval

  • Approved by the U.S. FDA (Tryvio; March 19, 2024).192

Forecasted revenue

  • Total global annual revenue forecasted to be $165 million by 2029.*

Sotatercept

(Winrevair)

Acceleron Pharma Inc.

  • Increased safety and efficacy
  • Novel mechanism
  • Breakthrough
  • Priority Review

Pulmonary arterial hypertension (PAH)

  • Clinical trials in Canada
  • Add-on Therapy

Approval

  • Approved by the U.S. FDA (Winrevair; March 26, 2024).193

Forecasted revenue

  • Total global annual revenue forecasted to be $5.6 billion by 2029.*

Dermatology

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Beremagene geperpavec

(Vyjuvek)

Krystal Biotech Inc.

  • Novel mechanism
  • Gene or cell therapy
  • Priority Review

Epidermolysis bullosa

  •  Biologic medicine

Approval

  • Approved by the U.S. FDA (Vyjuvek; May 19, 2023).194

Forecasted revenue

  • Total global annual revenue forecasted to be $895 million by 2029.*

Gastrointestinal disorders

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

RBX-2660

(Rebyota)

Ferring Pharmaceuticals Inc.

  • Increased safety and efficacy

Clostridium difficile infections (C. difficile associated disease)

  • Clinical trials in Canada

Approval

Approved by the U.S. FDA (Rebyota; November 30, 2023).195

Forecasted revenue

  • Forecasted annual global revenue unknown.

Resmetirom

(Rezdiffra)

Madrigal Pharmaceuticals Inc.

  • Increased safety and efficacy
  • Novel mechanism

Metabolic dysfunction-associated steatohepatitis (MASH)

  • Clinical trials in Canada

Approval

  • Approved by the U.S. FDA (Rezdiffra; March 14, 2024).196

Forecasted revenue

  • Total global annual revenue forecasted to be $3.9 billion by 2029.*

Genetic disorders

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Delandistrogene moxeparvovec

(Elevidys)

Sarepta Therapeutics Inc.

  • Novel mechanism
  • Gene or cell therapy
  • Priority Review

Duchenne muscular dystrophy

  • Biologic medicine

Approval

  • Approved by the U.S. FDA (Elevidys; June 22, 2023).197

Forecasted revenue

  • Total global annual revenue forecasted to be $3 billion by 2029.*

Hematological disorders

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Danicopan

(Voydeya)

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

Approval

  • Approved by the U.S. FDA (Voydeya; March 29, 2024). 198
  • Under review by Health Canada.199

Forecasted revenue

  • Total global annual revenue forecasted to be $302 million by 2029.*

Fidanacogene
elaparvovec

(Beqvez)

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

Approval

  • Approved by the U.S. FDA (Beqvez; April 25, 2024).200

Forecasted revenue

  • Total global annual revenue forecasted to be $516 million by 2029.*

Immunological disorders

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Omidubicel

(Omisirge)

Gamida Cell Ltd.

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

Hematopoietic stem cell transplantation

  • Rare or orphan designation
  • Biologic medicine

Approval

  • Approved by the U.S. FDA (Omisirge; April 17, 2023).201

Forecasted revenue

  • Forecasted annual global revenue unknown.

Metabolic Disorders

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Donislecel

(Lantidra)

CellTrans Inc.

  • Increased safety and efficacy

Type 1 diabetes (juvenile diabetes)

  • Biologic medicine

Approval

  • Approved by the U.S. FDA (Lantidra; June 28, 2023).202

Forecasted revenue

  • Forecasted annual global revenue unknown.

Insulin icodec

(Awiqli)

Novo Nordisk AS

  • Novel mechanism

Type 1 diabetes (juvenile diabetes); Type 2 diabetes

  • Clinical trials in Canada
  • Potential evergreening

Approval

  • Health Canada approved (Awiqli; March 12, 2024); not marketed as of June 17, 2024.203

Forecasted revenue

  • Total global annual revenue forecasted to be $1 billion by 2029.*

Pegunigalsidase alfa

(Elfabrio)

Chiesi Farmaceutici SpA

  • Increased safety and efficacy
  • Novel mechanism
  • Fast Track

Fabry disease

(FD)

  • Clinical trials in Canada
  • Rare or orphan designation

Approval

  • Approved by the U.S. FDA (Elfabrio; May 9, 2023).204
  • Approved by the EMA (Elfabrio; May 8, 2023).205

Forecasted revenue

  • Forecasted annual global revenue unknown.

Oncology

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Imetelstat sodium

(Rytelo)

Geron Corp

  • Increased safety and efficacy
  • Novel mechanism

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

  • Clinical trials in Canada

Approval

  • Approved by the U.S. FDA (Rytelo; June 6, 2024).206

Forecasted revenue

  • Total annual global revenue forecasted to be $1.2 billion by 2029.*

Ophthalmology

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Avacincaptad pegol sodium

(Izervay)

Astellas Pharma Inc.

  • Increased safety and efficacy
  • Fast Track

Geographic atrophy (GA)

  • Clinical trials in Canada

Approval

  • Approved by the U.S. FDA (Izervay; August 4, 2023).207

Forecasted revenue

  • Total global annual revenue forecasted to be $1.5 billion by 2029.*

Women’s health

Medicine (Trade name) Company Indication(s) Approval Status and Key Attributes

Fezolinetant

(Veozah)

Astellas Pharma Inc.

  • Increased safety and efficacy
  • Novel mechanism

Vasomotor symptoms of menopause (hot flashes)

  • Clinical trials in Canada

Approval

  • Approved by the U.S. FDA (Veozah; May 12, 2023).208
  • Approved by the EMA (Veozah; December 7, 2023).209

Forecasted revenue

  • Total global annual revenue forecasted to be $1.4 billion by 2029.*

* Consensus forecasts for global revenue data were collected from GlobalData, Q2-2024, 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.

Table 7 highlights six 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 five medicines reported in the 2022 edition, all but one have received market authorization from Health Canada. The central nervous system drug masitinib mesylate, indicated for amyotrophic lateral sclerosis (ALS), was issued a Notice of Deficiency (2024-02), and the submission was withdrawn 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, 2023

Immunological disorders

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

Leniolisib

Joenja (U.S.)

Pharming Technologies BV

  • Novel mechanism

Activated phosphoinositide 3-kinase delta syndrome (APDS)

  • Priority Review
  • Rare or orphan designation
  • It is a PI3K-δ selective inhibitor.210
  • Administered orally.
  • Approved by the U.S. FDA (Joenja tablets; March 24, 2023) for APDS.211

Clinical trials

  • Based on an interim analysis of an ongoing open-label, single-arm extension study, it was well tolerated and maintained durable outcomes with up to 5 years of exposure in 37 patients with APDS.212
  • No other therapy for APDS was identified in Phase II development at this time.213

Forecasted revenue

  • Total global annual revenue forecasted to be $260 million by 2029.*

Tapinarof

Vtama (U.S.)

Dermavant Sciences GmbH

  • Novel mechanism

Plaque psoriasis

  • It is a non-steroidal, topical, aryl hydrocarbon receptor (AhR) agonist.214
  • Administered topically once a day.
  • Approved by the U.S. FDA (Vtama; May 23, 2022) for the topical treatment of plaque psoriasis in adults.215

Clinical trials

  • Clinical trials demonstrated high rates of complete skin clearance with tapinarof cream, durable effects while on treatment (a lack of tachyphylaxis for up to 52 weeks), an approximately 4-month remittive effect off therapy after achieving complete clearance and stopping treatment (i.e., duration during which psoriasis does not recur off therapy), and no rebound effects after cessation of therapy.216
  • There are other drugs in Phase II development (n>10), but none with the same mechanism of action as tapinarof.  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $790 million by 2029.*

Oncology

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

Avapritinib

Ayvakit (U.S.)

Blueprint Medicines Corporation

  • Novel mechanism

Gastrointestinal stromal tumor (GIST)

  • Breakthrough
  • Fast Track
  • Rare or orphan designation
  • It is a selective tyrosine kinase inhibitor (TKI) targeting KIT D816V.
  • Administered orally.
  • Approved by the U.S. FDA (Ayvakit; tablets on June 16, 2021) for mastocytosis.217 Approved by the EMA (Ayvakit; September 30, 2020).218

Clinical trials

  • In a Phase II trial, it was superior to placebo in reducing uncontrolled symptoms and mast-cell burden in patients with indolent systemic mastocytosis.219
  • No other therapy for systemic mastocytosis was identified in Phase II development at this time. 220

Forecasted revenue

  • Forecasted annual global revenue $1.6 billion by 2029.*

Ivosidenib

Tibsovo (U.S.)

Servier Canada Inc.

  • Novel mechanism

Acute myeloid leukemia (AML)

  • Breakthrough
  • Priority Review
  • Rare or orphan designation
  • A It is a selective, allosteric, isocitrate dehydrogenases 1 and 2 (IDH1 and IDH2) inhibitor.221
  • Administered orally.
  • Approved by the U.S. FDA (Tibsovo; May 25, 2022) in combination with azacitidine (azacitidine for injection) for newly diagnosed AML with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.222  Approved by the EMA (Tibsovo; May 12, 2023).223

Clinical trials

  • Clinical data highlighted its favourable safety profile, as a stand-alone therapy and in combination strategy. 224
  • In a comparison of real-world outcomes with common first-line regimens for IDH1-mutated AML, ivosidenib plus a hypomethylating agent was associated with better outcomes than venetoclax plus a hypo-methylating agent.225
  • There are other drugs in Phase II development (n>10), but none with the same mechanism of action as ivosidenib.  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue $29 million by 2029.*

Momelotinib dihydrochloride monohydrate

Ojjaara (U.S.)

Omjjara (EMA)

GlaxoSmithKline Inc.

  • Increased safety and efficacy
  • Novel mechanism

Myelofibrosis (MF)

  • Fast Track
  • Rare or orphan designation
  • It is an inhibitor of Janus kinase 1 (JAK1) and JAK2 and also an inhibitor of activin A receptor type 1 (ACVR1), a key regulator of iron homeostasis.
  • Administered orally, once a day.
  • Approved by the U.S. FDA (Ojjaara; September 15, 2023) for myelofibrosis patients with anemia.226 Approved by the EMA (Omjjara; February 8, 2024).227

Clinical trials

  • Clinical trials have demonstrated its efficacy in reducing spleen size, alleviating symptoms, and improving anemia, with a favourable safety profile compared to other JAK inhibitors, both in treatment-naïve and in pre-treated patients.228, 229
  • In anemic subgroups, momelotinib was associated with higher rates of transfusion independence and reduced/stable transfusion intensity vs. ruxolitinib.230
  • In addition to these benefits, it conferred notable survival outcomes in both JAK inhibitor-naïve and ruxolitinib-pretreated patients.231
  • There are other drugs in Phase II development (n>10), but none with the same dual mechanism of action as momelotinib.  See Appendix A for additional information.

Forecasted revenue

  • Total global annual revenue forecasted to be $939 million by 2029.*

Ophthalmology

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

Perfluorohexyl-octane

Miebo (U.S.)

Bausch & Lomb Inc.

  • Novel mechanism

Keratoconjunctivitis sicca (dry eye)

  • It is a s a semifluorinated alkane. It forms a monolayer at the air-liquid interface of the tear film which can be expected to reduce evaporation.
  • Administered as topical ophthalmic drops.
  • Approved by the U.S. FDA (Miebo; May 19, 2023) for the treatment of dry eye disease.232

Clinical trials

  • Compared with use of hypotonic saline solution, instillation of perfluorohexyloctane led to significant improvements in signs and symptoms in as early as 2 weeks. In a long-term, open-label safety extension study, efficacy of perfluorohexyloctane was sustained over 12 months, and the safety profile was consistent with those of previous studies. Clinical trial results indicate that treatment with perfluorohexyloctane effectively and consistently reduces the signs and symptoms of DED.233
  • There are other drugs in Phase II development (n>10), but none with the same mechanism of action as perfluorohexyloctane.  See Appendix A for additional information.

Forecasted revenue

  • Forecasted annual global revenue unknown.

* Consensus forecasts for global revenue data were collected from GlobalData, Q2-2024, 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 CDA Reimbursement Review report.
Data source: GlobalData Healthcare database.

Appendix A

Table A1: Biosimilars in Phase III or preregistration (based on data extract from 2024-04-03)

Medicine Reference product in Canada Other biosimilars marketed in Canada at this time (Y/N) and biosimilars under review by HC Companies developing a biosimilar Indication

Aflibercept

Eylea

(Bayer Inc.)

N

Under HC review (as of Apr 3):

  • Amgen Canada Inc.
  • Apotex Inc.
  • Biosimilar Collaborations Ireland Ltd.
  • Celltrion HealthCare Co Ltd.

Alteogen Inc.

Celltrion Inc.

Formycon AG

Sam Chun Dang Pharm Co Ltd.

Wet (neovascular/exudative) macular degeneration

Amgen Inc.

Macular edema

Celltrion Inc.

Age related macular degeneration

Celltrion Inc.

Choroidal neovascularization

Celltrion Inc.

Cystoid macular edema; Diabetic macular edema

Bevacizumab

Avastin

(Hoffmann-La Roche Limited)

Y

Prestige BioPharma Ltd.

Non-small cell lung cancer

Denosumab

Prolia/Xgeva

(Amgen Canada Inc.)

N

Under HC review (as of Apr 3):

  • Sandoz Canada Inc. (approved 2024-02-16 but not yet marketed)

Alvotech SA

Biocon Ltd.

Celltrion Inc.

Eden Biologics Inc.

Fresenius Kabi SwissBioSim GmbH

Gedeon Richter plc

I'rom Group Co Ltd. (Japan)

Shanghai Henlius Biotech Inc. 

Post menopausal osteoporosis

Biocon Ltd.

Bone fracture

Biocon Ltd.

Bone metastasis

Eculizumab

Soliris

(Alexion Pharma GmbH)

N

Under HC review (as of Apr 3):

  • Amgen Canada Inc.
  • Samsung Bioepis Co., Ltd.
 

Paroxysmal nocturnal hemoglobinuria

Filgrastim

Neulasta

(Amgen Canada Inc.)

Y

Under HC review (as of Apr 3):

  • Curateq Biologics Private Limited

Chemotherapy induced neutropenia

Ocrelizumab

Ocrevus

(Hoffmann-La Roche Limited)

N

Celltrion Inc.

Relapsing remitting multiple sclerosis (RRMS)

Omalizumab

Xolair

(Novartis Pharmaceuticals Canada Inc.)

N

Under HC review (as of Apr 3):

  • Celltrion Healthcare Co Ltd.

Celltrion Inc.

Allergic asthma

Celltrion Inc.

Chronic urticaria or hives

Celltrion Inc.

Nasal polyps (nasal polyposis); Rhinosinusitis

Celltrion Inc.

Food allergy

Pegfilgrastim

Neulasta

(Amgen Canada Inc.)

Y

Under HC review (as of Apr 3):

  • Curateq Biologics Private Limited
  • JAMP Pharma Corporation
  • Lupin Pharma Canada Limited
  • Nora Pharma Inc.

Curateq Biologics Pvt Ltd.

Chemotherapy induced neutropenia

Pertuzumab

Perjeta

(Hoffmann-La Roche Limited)

N

Shanghai Henlius Biotech Inc.

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

Ranibizumab

Lucentis

(Novartis Pharmaceuticals Canada Inc.)

Y

Lupin Ltd.

Wet (neovascular/exudative) macular degeneration

Rituximab

Rituxan

(Hoffmann-La Roche Limited)

Y

Under HC review (as of Apr 3):

  • Dr. Reddy's Laboratories SA

Non-Hodgkin’s lymphoma;

chronic lymphocytic leukemia; rheumatoid arthritis; granulomatosis with polyangiitis and microscopic polyangiitis

Tocilizumab

Actemra

(Hoffmann-La Roche Limited)

N

Under HC review (as of Apr 3):

  • Fresenius Kabi Canada Ltd.

Celltrion Inc.

Mochida Pharmaceutical Co Ltd.

Rheumatoid arthritis

Trastuzumab

Herceptin

(Hoffmann-La Roche Limited)

Y

Under HC review (as of Apr 3):

  • Accord Healthcare Inc.

Prestige BioPharma Ltd.

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

Prestige BioPharma Ltd.

Adenocarcinoma of the gastroesophageal junction

Prestige BioPharma Ltd.

Gastric cancer

Ustekinumab

Stelara

(Janssen Inc.)

N

Under HC review (as of Apr 3):

  • Celltrion Healthcare Co Ltd.
  • Samsung Bioepis Co,. Ltd..

Biocon Ltd.

Samsung Bioepis Co Ltd.

Psoriatic arthritis

Biocon Ltd.

Formycon AG

Samsung Bioepis Co Ltd.

STgen Bio Co Ltd.

Plaque psoriasis (psoriasis vulgaris)

Biocon Ltd.

Samsung Bioepis Co Ltd.

Crohn's disease (regional enteritis)

Samsung Bioepis Co Ltd.

Ulcerative colitis

Table A2: Drugs in Phase II for indications targeted by pipeline candidates, 2023

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

ABBVCLS-7262

Amyotrophic lateral sclerosis (ALS)

Drugs in Phase II for this indication and their MoA :

  • 1 phosphatidylinositol 3 phosphate 5 kinase inhibitor (apilimod);
  • 70 kDa ribosomal protein S6 kinase inhibitor/serine/threonine protein kinase mTOR  inhibitor(monepantel);
  • Akt pathway activator (IPL-344, NA-704, RNS-60);
  • antioxidant (EH-301);
  • apoptosis regulator BAX Inhibitor/free radical scavenger/ insulin like growth factor I  and II activator/ insulin receptor agonist/protein kinase B activator (GM-6);
  • arachidonate 15 lipoxygenase inhibitor (utreloxastat);
  • ataxin 2 inhibitor (ION-541);
  • boosts regulatory T-cells (COYA-101);
  • CD40 ligand inhibitor (tegoprubart);
  • cell therapy (AstroRx);
  • cholinergic receptor muscarinic antagonist (scopolamine);
  • coagulation factor V and  factor VIII inhibitor (3K3A-APC);
  • combination (celecoxib + ciprofloxacin);
  • D2 and D3 dopamine receptor agonist (ropinirole);
  • gene therapy (Gene Therapy to Activate GDNF for Amyotrophic Lateral Sclerosis and Retinitis Pigmentosa);
  • glucocorticoid receptor antagonist (dazucorilant);
  • ikappaB kinase inhibitor / nuclear factor kappa B inhibitor (acetylcysteine zidrimer);
  • macrophage activator (NP-001);
  • macrophage colony stimulating factor 1 receptor antagonist (sotuletinib);
  • mitochondrial-targeted neuroprotector (MP-101);
  • oxidoreductase enzyme inhibitor (EPI-589);
  • protein phosphatase 1 regulatory subunit 15A  inhibitor (icerguastat);
  • reverse transcriptase inhibitor (censavudine);
  • superoxide dismutase  activator (AP-101);
  • others with undisclosed mechanism of action (TM-700).

There are no other drugs targeting this indication with the same MoA as ABBVCLS-7262 (a eukaryotic translation initiation factor 2 subunit beta activator) in Phase II development at this time.

 

AD109 (atomoxetine + R-oxybutynin)

Obstructive sleep apnea (OSA)

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

Aficamten

Hypertrophic cardiomyopathy

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

Brensocatib

Bronchiectasis

Drugs in Phase II for this indication and their MoA :

  • mitogen activated protein kinase inhibitor (MGS-2525);
  • neutrophil elastase  inhibitor (alvelestat);
  • synthetic phage (AP-PA02).

There are no other drugs targeting this indication with the same MoA as brensocatib (a dipeptidyl peptidase 1 inhibitor) in Phase II development at this time.

Datopotamab deruxtecan

Breast cancer, both HR-positive/HER2-negative and triple-negative

Drugs in Phase II for this indication and their MoA :

  • gene-modified cell therapy (huMNC2-CAR22, huMNC2-CAR44);
  • prolyl endopeptidase FAP inhibitor (AAA-614);
  • vaccine (nelipepimut-S).

There are no other drugs for all types of breast cancer targeted by datopotamab (a TROP2-targeted antibody-drug conjugate) in Phase II development at this time.

Efruxifermin

Metabolic dysfunction-associated steatohepatitis (MASH)

Drugs in Phase II for this indication and their MoA :

  • 1-acylglycerol 3 phosphate o acyltransferase PNPLA3 inhibitor (AZD-2693);
  • 5-lipoxygenase inhibitor (epeleuton);
  • acetyl CoA carboxylase inhibitor; diacylglycerol O acyltransferase 2 inhibitor (combination of clesacostat tromethamine + ervogastat);
  • AMP activated protein kinase activator; cGMP specific 3',5' cyclic PDE inhibitor; NAD dependent protein deacetylase sirtuin 1 activator (combination of leucine + metformin hydrochloride + sildenafil citrate);
  • androgen receptor agonist (LPCN-1144);
  • arachidonate 5 inhibitor; leukotriene receptor antagonist; PDE 3, 4 inhibitor; phospholipase C inhibitor; thromboxane A2 receptor antagonist (tipelukast);
  • bile acid receptor agonist (CS-0159, HPG-1860, TERN-101);
  • cell therapy (HepaStem);
  • cholesterol and triglyceride synthesis dual inhibitor (gemcabene);
  • cytochrome P450 2E1 inhibitor; diacylglycerol O acyltransferase 1 inhibitor; hormone sensitive lipase activator (SNP-610);
  • focal adhesion kinase inhibitor (narmafotinib);
  • GLP 1 receptor agonist; glucagon receptor agonist (AZD-9550, efinopegdutide, efocipegtrutide, pemvidutide);
  • glucose dependent insulinotropic receptor agonist (DA-1241);
  • high mobility group protein B1 activator (redasemtide trifluoroacetate);
  • lipid modulator (berberine ursodeoxycholate);
  • mitochondrial pyruvate carrier inhibitor (PXL-065);
  • peptidyl prolyl cis trans isomerase A, B & F mitochondrial inhibitor(rencofilstat);
  • thyroid hormone receptor beta agonist (ALG-009, TERN-501, VK-2809).

There is one other drug (NN-9499) targeting this indication with the same MoA as efruxifermin (a fibroblast growth factor receptor agonist) in Phase II development at this time. 

Ensifentrine

Chronic obstructive pulmonary disease

Drugs in Phase II for this indication and their MoA :

  • 5-lipoxygenase inhibitor (epeleuton);
  • adenosine receptor A1 antagonist (PBF-680);
  • beta 1 and 2 adrenergic receptor antagonist (nadolol);
  • complement C1q subcomponent inhibitor (RLS-0071);
  • macrophage metalloelastase inhibitor (aderamastat);
  • myristoylated alanine rich C kinase substrate inhibitor (BIO-11006);
  • toll like receptor 2, 6, 9 agonist (PUL-042);
  • vaccine, inactivated (HI-164OV).

There are no other drugs targeting this indication with the same MoA as ensifentrine (a PDE 3 and 4 inhibitor) in Phase II development at this time.

Fazirsiran

Alpha-1 antitrypsin deficiency (A1AD)

Drugs in Phase II for this indication and their MoA :

  • neutrophil elastase inhibitor (alvelestat).

There are other drugs (belcesiran, INBRX-101) targeting this indication with the same MoA as fazirsiran (an alpha 1 antitrypsin Inhibitor) in Phase II development at this time.

Gemcitabine (GemRIS, TAR-200)

Non-muscle invasive bladder cancer (NMIBC) (superficial bladder cancer); Muscle invasive bladder cancer (MIBC)

Drugs in Phase II for these indications and their MoA :

  • vaccine (lerapolturev).

There is no other drug targeting both indications with the same MoA as gemcitabine (a ribonucleoside diphosphate reductase large subunit inhibitor) in Phase II development at this time.

Iclepertin

Kidney transplant rejection

Drugs in Phase II for this indication and their MoA :

  • CD40 ligand inhibitor (tegoprubartFootnote viii);
  • critical regulator of the microtubule motor cytoplasmic dynein (LIS-1);
  • gene-modified cell therapy (TX-200);
  • T cell specific surface glycoprotein CD28 antagonist (FR-104);
  • T cell surface antigen CD2 inhibitor (siplizumab).

There is no other drug targeting this indication with the same MoA as iclepertin (a sodium and chloride dependent glycine transporter 1 inhibitor) in Phase II development at this time.

Inaxaplin

Focal segmental glomerulosclerosis

Drugs in Phase II for this indication and their MoA :

  • B lymphocyte antigen CD19 inhibitor (VB-119);
  • lipid modifier (R3R01).

There is no other drug targeting this indication with the same MoA as inaxaplin (an apolipoprotein L1 inhibitor) in Phase II development at this time.

Nerinetide

 

Acute ischemic stroke

Drugs in Phase II for this indication and their MoA :

  • antioxidant (LT-3001);
  • apoptosis regulator BAX inhibitor/ free radical scavenger/ insulin like growth factor I and II activator/ insulin receptor agonist/ protein kinase B activator (GM-6);
  • bifunctional epoxide hydrolase 2 inhibitor (JX-10);
  • cell therapy (it-hMSC, NCS-01, ReN-001);
  • high mobility group protein B1 activator (redasemtide);
  • oxygen carrier (perflenapent);
  • repulsive guidance molecule a inhibitor (elezanumab);
  • others with undisclosed mechanism of action (RNS-60).

There is no other drug targeting this indication with the same MoA as nerinetide (a neuroprotective eicosapeptide) in Phase II development at this time.

Obefazimod

Ulcerative colitis

Drugs in Phase II for this indication and their MoA :

  • adenosine receptor A1/A3 antagonist (PBF-677);
  • bacteria replacement/microbiome modulator (BGP-014, VE-202A);
  • calcineurin inhibitor (cyclosporine CR);
  • cAMP specific 3',5' cyclic PDE 4B/4D inhibitor (orismilast);
  • E3 ubiquitin protein ligase pellino homolog 1 inhibitor (BBT-401);
  • integrin alpha 4/ beta 7 antagonist (MORF-057);
  • interleukin 1 alpha/ beta inhibitor (lutikizumab);
  • interleukin 2 receptor agonist (PT-101);
  • interleukin 7 receptor subunit alpha antagonist (lusvertikimab);
  • lanC like protein 2 agonist (omilancor);
  • melanocyte stimulating hormone receptor agonist (PL-8177);
  • microbial-derived immunotherapy (QBECOFootnote ix);
  • neuroimmune modulator (HM-201);
  • prostaglandin G/H synthase inhibitor (combination of mesalamine + hyaluronate sodium);
  • P selectin glycoprotein ligand 1 activator (AbGn-268);
  • sphingosine 1-phosphate receptor 1 agonist (amiselimod, icanbelimod);
  • toll like receptor 9 agonist (cobitolimod);
  • TNF ligand superfamily member 15 inhibitor (RG-6631);
  • others with undisclosed mechanism of action (KSP-0243).

There is no other drug targeting this indication with the same MoA as obefazimod (a eukaryotic translation initiation factor 4E inhibitor/ interleukin 22 receptor agonist) in Phase II development at this time.

Patidegib

Gorlin syndrome (basal cell nevus syndrome/nevoid basal cell carcinoma syndrome)

Drugs in Phase II for this indication and their MoA :

  • serine/threonine protein kinase mTOR inhibitor (sirolimus).

There is no other drug targeting this indication with the same MoA as patidegib (a hedgehog signaling pathway blocker) in Phase II development at this time.

Pelacarsen sodium 

Cardiovascular disease;  Hyperlipidemia

Drugs in Phase II for this indication and their MoA :

  • angiopoietin related protein 3  inhibitor (solbinsiran, zodasiran);
  • cholesterol and triglyceride synthesis dual inhibitor (gemcabene);
  • Niemann Pick C1 like protein 1 inhibitor (KT-6971);
  • proprotein convertase subtilisin/kexin type 9 inhibitor (AZD-0780).

There is no other drug targeting the hyperlipidemia indication with the same MoA as pelacarsen (an apolipoprotein A inhibitor) in Phase II development at this time.

Prademagene zamikeracel

Epidermolysis bullosa

Drugs in Phase II for this indication and their MoA :

  • bacterial biofilm/growth inhibitor / intermediate filament protein modulator (Tolasure);
  • high mobility group protein B1 activator (redasemtide trifluoroacetate);
  • others with undisclosed mechanism of action (RLF-TD011).

There is one drug (PTR-01) targeting this indication with the same MoA as prademagene zamikeracel (a collagen type 7 replacement) in Phase II development at this time.

Resiniferatoxin

Osteoarthritis pain

Drugs in Phase II for this indication and their MoA :

  • adenosine receptor A3 agonist (NTM-006);
  • C-C chemokine receptor type 2 antagonist (CNTX-6970);
  • C-C motif chemokine 17 inhibitor (GSK-3858279);
  • glucocorticoid receptor agonist (fluticasone);
  • high affinity nerve growth factor receptor inhibitor (AK-1830);
  • PG E2 receptor EP4 subtype antagonist (grapiprant);
  • PG G/H synthase 1 and 2  inhibitor (otenaproxesul);
  • sodium channel protein type 10 subunit alpha blocker (VX-150);
  • others with undisclosed mechanism of action (S-120083, TTAX-03).

There is one other drug (CGS-2005) targeting this indication with the same MoA as resiniferatoxin (a transient receptor potential cation channel subfamily v member 1 activator) in Phase II development at this time.

Revumenib

Acute lymphocytic leukemia (ALL); Acute lympho-blastic leukemia;

Refractory acute myeloid leukemia;

Relapsed acute myeloid leukemia

Drugs in Phase II for these indications and their MoA :

  • anti-CD123 antibody alkylating agent (pivekimab sunirine);
  • butyrophilin subfamily 3 member A1 activator (ICT-01);
  • cell therapy (ECT-001, SMART-101);
  • protein kinase B inhibitor (PTX-200).

There are other drugs (DS-1594b, DSP-5336, ziftomenib) targeting all indications with the same MoA as revumenib (a menin-mixed lineage leukemia inhibitor) in Phase II development at this time.

RGX-121

Mucopolysaccharidosis II (MPS II) (Hurler syndrome)

Drugs in Phase II for this indication and their MoA :

  • alpha L-iduronidase replacement (AGT-181, lepunafusp alfa);
  • CCAAT/enhancer binding protein alpha activator (MTL-CEBPA);
  • chondrocyte metabolism stimulator (pentosan polysulfate sodium).

There is one other drug (RGX-111) targeting this indication with the same MoA as RGX-121 (an iduronate 2 sulfatase activator) in Phase II development at this time.

Xanomeline-trospium (KarXT)

Schizophrenia; Psychosis

Drugs in Phase II for this indication and their MoA :

  • 5-hydroxytryptamine receptor 2A antagonist / D2 dopamine receptor antagonist (risperidone 6-12 month formulation).

There are other drugs (ANAVEX-371, NBI-1117568, RL-007) targeting the schizophrenia indication with the same MoA as xanomeline-trospium (a muscarinic acetylcholine receptor agonist) in Phase II development at this time.

Abbreviations:
AMP: adenosine monophosphate; GABA: gamma-aminobutyric acid ; GLP: glucagon like peptide; GSK: glycogen synthase kinase; PDE: phosphodiesterase; PG: prostaglandin; PPAR: peroxisome proliferator activated receptor; TNF: tumour necrosis factor.

* Data source: GlobalData’s Healthcare database (accessed April 2024).

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  65. A Randomized, Double-blind, Placebo-controlled, Multicenter Phase III Study to Evaluate the Efficacy and Safety of ABX464 Once Daily for Induction Treatment in Subjects With Moderately to Severely Active Ulcerative Colitis. ClinicalTrials.gov: NCT05507203 (recruiting). https://clinicaltrials.gov/study/NCT05507203?intr=obefazimod&aggFilters=phase:3&rank=3
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  68. A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F1 Fibrosis. ClinicalTrials.gov: NCT06165341 (recruiting). https://clinicaltrials.gov/study/NCT06165341?intr=Fazirsiran%20&aggFilters=phase:3&rank=2
  69. A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis. ClinicalTrials.gov: NCT05677971 (recruiting). https://clinicaltrials.gov/study/NCT05677971?intr=Fazirsiran%20&aggFilters=phase:3&rank=3
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  75. A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome). ClinicalTrials.gov: NCT03566043 (active, not recruiting). https://clinicaltrials.gov/study/NCT03566043?intr=RGX-121&aggFilters=phase:3&rank=1
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  79. AstraZeneca. Press Release, 2024. Datopotamab deruxtecan Biologics License Application accepted in the US for patients with previously treated metastatic HR-positive, HER2-negative breast cancer. 2 April 2024. https://www.astrazeneca.com/media-centre/press-releases/2024/fda-accepts-dato-dxd-bla-for-breast-cancer.html
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  81. A Phase 3, Open-label, Randomised Study of Datopotamab Deruxtecan (Dato-DXd) Versus Investigator's Choice of Chemotherapy in Patients Who Are Not Candidates for PD-1/PD-L1 Inhibitor Therapy in First-line Locally Recurrent Inoperable or Metastatic Triple-negative Breast Cancer (TROPION Breast02). ClinicalTrials.gov: NCT05374512 (recruiting). https://clinicaltrials.gov/study/NCT05374512?intr=Datopotamab%20&aggFilters=phase:3&rank=6
  82. A Phase III, Open-label, Randomised Study of Datopotamab Deruxtecan (Dato-DXd) With or Without Durvalumab Compared With Investigator's Choice of Chemotherapy (Paclitaxel, Nab-paclitaxel or Gemcitabine + Carboplatin) in Combination With Pembrolizumab in Patients With PD-L1 Positive Locally Recurrent Inoperable or Metastatic Triple-negative Breast Cancer (TROPION-Breast05). ClinicalTrials.gov: NCT06103864 (recruiting). https://clinicaltrials.gov/study/NCT06103864?intr=Datopotamab%20&aggFilters=phase:3&rank=8
  83. A Phase-3, Open-Label, Randomized Study of Dato-DXd Versus Investigator's Choice of Chemotherapy (ICC) in Participants With Inoperable or Metastatic HR-Positive, HER2-Negative Breast Cancer Who Have Been Treated With One or Two Prior Lines of Systemic Chemotherapy (TROPION-Breast01). ClinicalTrials.gov: NCT05104866 (active, not recruiting). https://clinicaltrials.gov/study/NCT05104866?intr=Datopotamab%20&aggFilters=phase:3&rank=9
  84. A Phase 3 Open-label, Randomised Study of Datopotamab Deruxtecan (DatoDXd) With or Without Durvalumab Versus Investigator's Choice of Therapy in Patients With Stage I-III Triple-negative Breast Cancer Who Have Residual Invasive Disease in the Breast and/or Axillary Lymph Nodes at Surgical Resection Following Neoadjuvant Systemic Therapy (TROPION-Breast03). ClinicalTrials.gov: NCT05629585 (recruiting). https://clinicaltrials.gov/study/NCT05629585?intr=Datopotamab%20&aggFilters=phase:3&rank=10
  85. A Phase III, Open-label, Randomised Study of Neoadjuvant Datopotamab Deruxtecan (Dato-DXd) Plus Durvalumab Followed by Adjuvant Durvalumab With or Without Chemotherapy Versus Neoadjuvant Pembrolizumab Plus Chemotherapy Followed by Adjuvant Pembrolizumab With or Without Chemotherapy for the Treatment of Adult Patients With Previously Untreated Triple-Negative or Hormone Receptor-low/HER2-negative Breast Cancer (D926QC00001; TROPION-Breast04). ClinicalTrials.gov: NCT06112379 (recruiting). https://clinicaltrials.gov/study/NCT06112379?intr=Datopotamab%20&aggFilters=phase:3&page=2&rank=11
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  90. A Phase 3, Open-Label, Multi-Center, Randomized Study Evaluating the Efficacy and Safety of TAR-200 in Combination With Cetrelimab or TAR-200 Alone Versus Intravesical Bacillus Calmette-Guérin (BCG) in Participants With BCG-naïve High-Risk Non-Muscle Invasive Bladder Cancer. ClinicalTrials.gov: NCT05714202 (recruiting). https://clinicaltrials.gov/study/NCT05714202?intr=TAR-200&aggFilters=phase:3&rank=1
  91. A Phase 3, Randomized, Open-label, Multi-center Study Evaluating the Efficacy and Safety of TAR-200 Versus Investigator's Choice of Intravesical Chemotherapy in Participants Who Received Bacillus Calmette-Guérin (BCG) and Recurred With High-risk Non-muscle-invasive Bladder Cancer (HR-NMIBC) and Who Are Ineligible for or Elected Not to Undergo Radical Cystectomy. ClinicalTrials.gov: NCT06211764 (recruiting). https://clinicaltrials.gov/study/NCT06211764?intr=TAR-200&aggFilters=phase:3&rank=2
  92. A Phase 3, Multi-center, Randomized Study Evaluating Efficacy of TAR-200 in Combination With Cetrelimab Versus Concurrent Chemoradiotherapy in Participants With Muscle-Invasive Urothelial Carcinoma (MIBC) of the Bladder Who Are Not Receiving Radical Cystectomy. ClinicalTrials.gov: NCT04658862 (recruiting). https://clinicaltrials.gov/study/NCT04658862?intr=TAR-200&aggFilters=phase:3&rank=3
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  95. A Multicenter, Randomized, Double-blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome. ClinicalTrials.gov: NCT03703310 (completed). https://clinicaltrials.gov/study/NCT03703310?intr=patidegib%20&aggFilters=phase:3&rank=2
  96. A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Gorlin Syndrome. ClinicalTrials.gov: NCT06050122 (recruiting). https://clinicaltrials.gov/study/NCT06050122?intr=patidegib%20&aggFilters=phase:3&rank=1
  97. A Phase 3, Multicenter, Open-Label Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome). ClinicalTrials.gov: NCT04308395 (terminated). https://clinicaltrials.gov/study/NCT04308395?intr=patidegib%20&aggFilters=phase:3&rank=3
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  102. Corcept Therapeutics to Start Phase 3 Trial of Relacorilant Plus Nab-Paclitaxel in Patients With Platinum-Resistant Ovarian Cancer. June 2, 2022. https://www.globenewswire.com/en/news-release/2022/06/06/2456669/0/en/Corcept-Therapeutics-to-Start-Phase-3-Trial-of-Relacorilant-Plus-Nab-Paclitaxel-in-Patients-With-Platinum-Resistant-Ovarian-Cancer.html
  103. A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study of AG-881 in Subjects With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation. ClinicalTrials.gov: NCT04164901 (active, not recruiting). https://clinicaltrials.gov/study/NCT04164901?intr=Vorasidenib%20&aggFilters=phase:3&rank=1
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  110. Phase 3 Randomized Double-Blind Placebo-Controlled 6-month Parallel-Arm Study to Compare a Fixed Dose Combination of Aroxybutynin/Atomoxetine (AD109) to Placebo in Obstructive Sleep Apnea (SynAIRgy Study). ClinicalTrials.gov: NCT05813275 (recruiting). https://www.clinicaltrials.gov/study/NCT05813275?intr=AD109&aggFilters=phase:3&rank=2
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  114. A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib Administered Once Daily for 52 Weeks in Subjects With Non-Cystic Fibrosis Bronchiectasis - The ASPEN Study. ClinicalTrials.gov: NCT04594369 (active, not recruiting). https://clinicaltrials.gov/study/NCT04594369?intr=Brensocatib&aggFilters=phase:3&rank=1
  115. A Randomised Double-blind Placebo-controlled Trial of Brensocatib (INS1007) in Patients With Severe COVID-19. ClinicalTrials.gov: NCT04817332 (completed). https://clinicaltrials.gov/study/NCT04817332?intr=Brensocatib&aggFilters=phase:3&rank=2
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  118. A Multicenter, Randomized, Open-label, Blinded Endpoint Evaluation, Phase 3 Study Comparing the Effect of Abelacimab Relative to Apixaban on Venous Thromboembolism (VTE) Recurrence and Bleeding in Patients With Cancer Associated VTE. ClinicalTrials.gov: NCT05171049 (recruiting). https://clinicaltrials.gov/ct2/show/NCT05171049?term=Abelacimab&draw=2&rank=2
  119. A Multicenter, Randomized, Open-label, Blinded Endpoint Evaluation, Phase 3 Study Comparing the Effect of Abelacimab vs. Dalteparin on Venous Thromboembolism (VTE) Recurrence and Bleeding in Patients With GI/GU Associated VTE. ClinicalTrials.gov: NCT05171075 (recruiting). https://clinicaltrials.gov/ct2/show/NCT05171075?term=Abelacimab&draw=2&rank=3 
  120. A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to evaLuate the effIcacy and Safety of abeLacimab in High-risk Patients With Atrial Fibrillation Who Have Been Deemed Unsuitable for Oral antiCoagulation (LILAC-TIMI 76). ClinicalTrials.gov: NCT05712200 (recruiting). https://clinicaltrials.gov/study/NCT05712200?intr=Abelacimab&aggFilters=phase:3&rank=1
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  122. Multi-Centre, Randomized, Double-Blind, Placebo-Controlled, Efficacy, and Safety Study of Etripamil Nasal Spray for the Termination of Spontaneous Episodes of Paroxysmal Supraventricular Tachycardia. NODE 301 Trial. ClinicalTrials.gov: NCT03464019 (completed). https://clinicaltrials.gov/ct2/show/NCT03464019?term=Etripamil&phase=2&draw=2&rank=5
  123. The NODE-303 Study: Multi-Centre, Multi-National,Open Label, Safety Study of Etripamil Nasal Spray for Patients With Paroxysmal Supraventricular Tachycardia. ClinicalTrials.gov: NCT04072835 (completed). https://clinicaltrials.gov/ct2/show/NCT04072835?term=Etripamil&phase=2&draw=2&rank=1
  124. Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Etripamil Nasal Spray Self-Administration for the Termination of Spontaneous Episodes of Paroxysmal Supraventricular Tachycardia in Chinese Patients. ClinicalTrials.gov: NCT05410860 (recruiting). https://clinicaltrials.gov/ct2/show/NCT05410860?term=Etripamil&phase=2&draw=2&rank=3
  125. An Open Label Extension Study of Etripamil Nasal Spray in Patients With Paroxysmal Supraventricular Tachycardia. ClinicalTrials.gov: NCT04952610 (Enrolling by invitation). https://clinicaltrials.gov/ct2/show/NCT04952610?term=Etripamil&phase=2&draw=2&rank=2
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  127. Rehman WU, Yarkoni M, Ilyas MA, Athar F, Javaid M, et al.  Cholesteryl Ester Transfer Protein Inhibitors and Cardiovascular Outcomes: A Systematic Review and Meta-Analysis. J Cardiovasc Dev Dis. 2024 May 16;11(5):152. doi: 10.3390/jcdd11050152.
  128. A Placebo-Controlled, Double-Blind, Randomized, Phase 3 Study to Evaluate the Effect of 10 mg Obicetrapib in Participants With a History of HeFH Who Are Not Adequately Controlled by Their Lipid Modifying Therapies. ClinicalTrials.gov: NCT05425745 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT05425745?term=Obicetrapib&phase=2&draw=2&rank=1
  129. A Placebo-Controlled, Double-Blind, Randomized Phase 3 Study to Evaluate the Effect of 10mg Obicetrapib in Participants With HeFH and/or ASCVD Who Are Not Adequately Controlled by Their Lipid Modifying Therapies. ClinicalTrials.gov: NCT05142722 (active, not recruiting). https://clinicaltrials.gov/ct2/show/NCT05142722?term=Obicetrapib&phase=2&draw=2&rank=2
  130. Placebo Controlled, Double Blind, Randomized Cardiovascular Outcome Study to Evaluate the Effect of 10 mg Obicetrapib in Participants With ASCVD Not Adequately Controlled Despite Maximally Tolerated Lipid Modifying Therapies. ClinicalTrials.gov: NCT05202509 (recruiting). https://clinicaltrials.gov/ct2/show/NCT05202509?term=Obicetrapib&phase=2&draw=2&rank=3
  131. A Placebo-Controlled, Double-Blind, Randomized, Phase 3 Study to Evaluate the Effect of Obicetrapib 10 mg and Ezetimibe 10 mg Fixed Dose Combination Daily on Top of Maximally Tolerated Lipid-Modifying Therapy in Participants With Heterozygous Familial Hypercholesterolemia (HeFH) and/or Atherosclerotic Cardiovascular Disease (ASCVD) or Multiple ASCVD Risk Factors. ClinicalTrials.gov: NCT06005597 (recruiting). https://clinicaltrials.gov/study/NCT06005597?intr=Obicetrapib&aggFilters=phase:3&rank=4
  132. A Placebo-controlled, Double-blind, Randomized, Phase 3 Study to Evaluate the Effect of Obicetrapib/Ezetimibe Fixed Dose Combination Daily on Coronary Plaque Characteristics in Participants With Atherosclerotic Cardiovascular Disease on Coronary CT Angiography (REMBRANDT Trial). ClinicalTrials.gov: NCT06305559 (not yet recruiting). https://clinicaltrials.gov/study/NCT06305559?intr=Obicetrapib&aggFilters=phase:3&rank=5
  133. A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adult Subjects With Dravet Syndrome (DS). ClinicalTrials.gov: NCT04940624 (completed). https://clinicaltrials.gov/ct2/show/NCT04940624?term=Soticlestat&phase=2&draw=2&rank=2
  134. A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Adult Subjects With Lennox-Gastaut Syndrome (LGS). ClinicalTrials.gov: NCT04938427 (completed). https://clinicaltrials.gov/ct2/show/NCT04938427?term=Soticlestat&phase=2&draw=2&rank=3
  135. A Phase 3, Prospective, Open-Label, Multisite, Extension of Phase 3 Studies To Assess the Long-Term Safety and Tolerability of Soticlestat as Adjunctive Therapy in Subjects With Dravet Syndrome or Lennox-Gastaut Syndrome (ENDYMION 2). ClinicalTrials.gov: NCT05163314 (recruiting). https://clinicaltrials.gov/ct2/show/NCT05163314?term=Soticlestat&phase=2&draw=2&rank=1
  136. An Open-label, Nonrandomized, Phase 3 Study to Evaluate the Efficacy and Safety of Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine. ClinicalTrials.gov: NCT06422377 (recruiting). https://clinicaltrials.gov/study/NCT06422377?intr=Soticlestat&aggFilters=phase:3&rank=1 
  137. Meglio M. 2023. Latozinemab Shows No Impact on Disease Progression in C9orf72 Frontotemporal Dementia Despite increasing Progranulin Expression. 1 November, 2023. https://www.neurologylive.com/view/latozinemab-shows-no-impact-disease-progression-c9orf72-frontotemporal-dementia-increasing-progranulin-expression
  138. A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene. ClinicalTrials.gov: NCT04374136 (active, not recruiting).  https://clinicaltrials.gov/ct2/show/NCT04374136?term=AL001&phase=2&draw=2&rank=1
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