Pfizer Announces FDA Approval of Beqvez Gene Therapy for Hemophilia B

Pfizer today announced that the FDA has approved Beqvez (fidanacogene elaparvovec-wyyy) for the treatment of adults with moderate-to-severe hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. Beqvez is an adeno-associated virus (AAV) vector-based gene therapy that delivers a functional copy of the Factor IX gene. In the Phase 3 BENEGENE-2 trial, Beqvez demonstrated a 71% reduction in the annualized bleeding rate compared to the lead-in period. This approval provides eligible patients with hemophilia B a one-time treatment option.