Review: Highlighting Five Critical FDA-approved Therapies of 2023

The evolution of regulatory adaptability and advancements in cell and gene therapy pipelines led the FDA to approve 35 new drugs in the first half of 2023. Notably, the Center for Drug Evaluation and Research (CDER) approved 26 of these, marking an increase of 10 from the same period the previous year. However, the FDA's Center for Biologics Evaluation and Research (CBER) exhibited exceptional activity.

Recent reports indicate that CBER granted approval to nine groundbreaking therapies within the first half of the year. This number surpasses the total approvals for any full year over the past two decades. Notable upcoming approvals for the latter half of 2023 include Iovance's Lifileucel, BrainStorm Cell Therapeutics' NurOwn, and a duo of gene therapies targeting sickle cell disease.

This article focuses on five significant therapies greenlit this year:

1.   Roctavian by BioMarin: CBER approved a third of its drugs for gene therapies in the first half. Roctavian addresses adults diagnosed with type A hemophilia, verified through FDA-endorsed tests to lack antibodies to serotype 5 adeno-associated virus. This pioneering gene therapy enters the market alongside other treatments, such as Roche's Hemlibra, a bispecific antibody which garnered over $4.2 billion in sales the previous year. Roctavian, priced at $2.9 million, ranks among the world's priciest drugs, shadowed only by Hemgenix at $3.5 million.

2.    Lantidra by CellTrans: In an embodiment of its penchant for innovation, the FDA granted approval to the inaugural cell therapy for type 1 diabetes (T1D) – Lantidra. Chicago-based CellTrans developed this islet cell therapy from the pancreatic cells of deceased donors. This therapy targets adult T1D patients struggling with recurrent hypoglycemia and challenges in maintaining target glucose levels. Several analogous therapies are in the pipeline, with Vertex boasting two mid-stage products and Sernova's Cell Pouch System in Phase I/II trials. Despite the buzz, challenges like immunosuppressive therapy toxicity and material scarcity persist.

3.    Elevidys by Sarepta: 2023 witnessed Sarepta's Elevidys becoming the inaugural gene therapy for Duchenne muscular dystrophy (DMD). Although the approval stirred debate, Sarepta clinched a narrow usage label for children aged 4-5.

4.    Arexvy by GlaxoSmithKline (GSK): Following extended anticipation, GSK launched its Arexvy vaccine in the US this May, marking the debut RSV vaccine. Designed to prevent RSV-induced lower respiratory tract disease in adults aged 60 and up, this vaccine addresses a virus typically mild in nature but potentially severe in infants and the elderly. GSK's approval precedes Pfizer's Abrysvo by less than a month. Analysts suggest that the surge in RSV cases the previous year potentially represents a market worth $5 billion to $10 billion.

5.   Omisirge by Gamida Cell: Hematopoietic stem cell transplantation (HCT), recognized as the paramount therapy for blood cancer patients, exacts a considerable physiological toll. Patients typically undergo intensive treatments to eradicate existing stem cells before HCT, elevating their infection risk—sometimes with lethal outcomes. Gamida Cell's Omisirge, extracted from umbilical cord blood, enhances and expands the cell's metabolic capacity. Post-processing, these cells are utilized in bone marrow transplants, fortifying the immune system and curtailing infection risks. Cord blood doesn't necessitate as rigorous matching as adult donor cells, potentially benefiting diverse racial or ethnic backgrounds who might not find an exact donor match.

Article Source: Bioon.com