FDA Accepts Pfizer’s Review Application for Hemophilia B Gene Therapy Treatment

Pfizer Inc. announced today that the U.S. Food and Drug Administration has accepted for review Pfizer’s Biologics License Application for fidanacogene elaparvovec for the treatment of adults with hemophilia B.

Fidanacogene elaparvovec is a novel, investigational gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX gene. For people living with hemophilia B, the goal of this gene therapy is to enable them to produce FIX themselves via this one-time treatment rather than needing regular intravenous infusions of FIX, as is the current standard of care.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date in the second quarter of 2024. Fidanacogene elaparvovec has been granted Breakthrough, Regenerative Medicines Advanced Therapy (RMAT) and orphan drug designations from the FDA.

Pfizer currently has three Phase 3 programs investigating gene therapy in populations where there is a high unmet need: hemophilia B, hemophilia A, and Duchenne muscular dystrophy. A Phase 3 trial is also ongoing investigating marstacimab, a novel, investigational anti-tissue factor pathway inhibitor (anti-TFPI) being studied for the treatment of people with hemophilia A and B with and without inhibitors.

For the full press release from Pfizer, click here.