Pfizer Receives FDA Approval for Beqvez Gene Therapy in Hemophilia B

Pfizer today announced that the FDA has approved Beqvez (fidanacogene elaparvovec) as a one-time gene therapy for the treatment of adults with moderately severe to severe hemophilia B who currently use Factor IX prophylaxis therapy. In the Phase 3 BENEGENE-2 trial, Beqvez demonstrated a 71% reduction in annualized bleeding rate compared to prior Factor IX prophylaxis. This approval represents a significant advancement in hemophilia B treatment, offering patients the potential for long-term bleed protection from a single administration.