New Medicines Approved in 2023 - Meds Entry Watch

Prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP)²

To treat geographic atrophy secondary to age-related macular degeneration²

Indicated for treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene²

To improve glycemic control in adults with type 2 diabetes mellitus as an adjunct to diet and exercise²

To treat breast cancer that meets certain disease criteria²

To treat anemia caused by chronic kidney disease for adults on dialysis for at least four months²

Indicated for treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene²

Indicated for the treatment of adults with type 1 diabetes who are unable to approach target HbA1C because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education²

To treat hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex²

Indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for: (1) routine prophylaxis to reduce the frequency of bleeding episodes; (2) on-demand treatment and control of bleeding episodes; and (3) perioperative management of bleeding²

To treat neutropenia²

To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated, advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy²

To treat adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy²

To treat relapsed or refractory diffuse large B-cell lymphoma (not otherwise specified) and high-grade B-cell lymphoma after two or more lines of systemic therapy²

To treat moderately to severely active ulcerative colitis in adults²

Indicated for treatment of sickle cell disease (SCD) in patients 12 years of age or older with recurrent vaso-occlusive crises (VOCs) ²

To prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI)²

To treat moderate to severe hot flashes caused by menopause²

To treat refractory, metastatic colorectal cancer²

To treat major depressive disorder²

To treat paroxysmal nocturnal hemoglobinuria²

To treat Alzheimer’s disease²

To treat activated phosphoinositide 3-kinase delta syndrome²

To treat Demodex blepharitis²

Indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events (VOEs)²

To treat intermediate or high-risk myelofibrosis in adults with anemia²

To use with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma²

To lower urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function²

To treat adults with progressing desmoid tumors who require systemic treatment²

To treat Friedrich’s ataxia²

Indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection²

To treat signs and symptoms of dry eye disease²

To treat relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor²

To treat patients 1 year old and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease²

To use as part of a treatment regimen for newly diagnosed acute myeloid leukemia that meets certain criteria²

To treat ROS1-positive non-small cell lung cancer²

To treat metastatic or recurrent locally advanced Merkel cell carcinoma²

To treat candidemia and invasive candidiasis²

To treat severely patchy hair loss²

To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive ²

To reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression²

To treat adults with relapsed or refractory multiple myeloma who have received at least four prior therapies²

To treat amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation²

To treat recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies²

To treat Rett syndrome²

Indicated for active immunization for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older²

To treat Duchenne muscular dystrophy²

To treat migraine²

To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive²

To treat postpartum depression²