Pfizer announces approval of BEQVE by FDA for adults with hemophilia B

Pfizer announced that the FDA has approved BEQVE, or fidanacogene elaparvovec-dzkt, for the treatment of adults with moderate to severe hemophilia B who currently use factor IX, or FIX, prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var, or AAVRh74var, capsid as detected by an FDA-approved test. BEQVEZ is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month. Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others. With BEQVEZ now approved for use, Pfizer is launching an innovative warranty program based on durability of patient response to treatment. The goal of the warranty is to provide greater certainty to payers, maximize access for eligible patients who receive BEQVEZ, and offer financial protection by insuring against the risk of efficacy failure. BEQVEZ is currently under review with the European Medicines Agency, and the treatment recently received regulatory approval in Canada.