Pfizer Receives FDA Approval for Beqvez Gene Therapy in Hemophilia A

Pfizer today announced that the FDA has approved Beqvez (giroctocogene fitelparvovec) for the treatment of adults with moderate-to-severe hemophilia A without inhibitors who currently use prophylactic factor VIII therapy. In the Phase 3 AFFINE trial, Beqvez demonstrated a statistically significant reduction in the annualized bleeding rate compared to prior prophylaxis, with 84% of participants having no treated bleeds at 15 months. Beqvez is an AAV gene therapy that delivers a functional copy of the factor VIII gene to liver cells. This approval provides eligible hemophilia A patients a one-time gene therapy option.