FDA Approves Pfizer's Gene Therapy for Hemophilia B

The U.S. Food and Drug Administration (FDA) has approved Pfizer Inc.'s gene therapy, BEQVEZ™ (fidanacogene elaparvovec-dzkt), for the treatment of adults with hemophilia B. This one-time treatment is designed to enable patients to produce their own clotting factor, potentially reducing bleeding events and the burden of regular infusions.

BEQVEZ is indicated for adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis or have experienced severe bleeding episodes. The therapy aims to allow the body to produce FIX internally, offering an alternative to the current standard of care which requires frequent intravenous infusions.

Hemophilia B is a rare genetic bleeding disorder characterized by a deficiency in FIX, leading to prolonged and frequent bleeding. Globally, over 38,000 people live with hemophilia B. Despite current prophylactic treatments, many individuals still face spontaneous bleeding and a significant treatment burden.

Pfizer is introducing a warranty program for BEQVEZ to provide greater certainty to payers and ensure patient access. The therapy has also received regulatory approval in Canada and is under review by the European Medicines Agency (EMA).