Pfizer’s application for hemophilia B gene therapy accepted by FDA

Pfizer announced that the FDA has accepted the company’s Biologics License Application – BLA – for fidanacogene elaparvovec for the treatment of adults with hemophilia B. In parallel, the European marketing authorization application for fidanacogene elaparvovec has also been accepted and is under review by the EMA. Fidanacogene elaparvovec is a novel, investigational gene therapy that contains a bio-engineered adeno-associated virus capsid and a high-activity variant of human coagulation Factor IX – FIX – 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 Phase 3 BENEGENE-2 study, which met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with FIX, administered as part of usual care. Fidanacogene elaparvovec was generally well-tolerated, with a safety profile consistent with Phase 1/2 results. The FDA has set a Prescription Drug User Fee Act goal date in 2Q24. Fidanacogene elaparvovec has been granted Breakthrough, Regenerative Medicines Advanced Therapy and orphan drug designations from the FDA.