Pfizer's Beqvez Gets FDA Nod
- FDA approval boosts Pfizer's gene therapy portfolio
- Beqvez warranty program provides financial protection
- Pfizer to monitor Beqvez's long-term durability for 15 years
Pfizer has received FDA approval for Beqvez, a gene therapy for certain patients with moderate to severe hemophilia B. Hemophilia B is a genetic bleeding disorder that prevents normal blood clotting. Beqvez is designed to enable people living with this condition to produce factor IX themselves, eliminating the need for regular intravenous infusions.
The approval covers the treatment of adults who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and don't have neutralizing antibodies to adeno-associated virus serotype Rh74var. The approval was based on the BENEGENE-2 Phase 3A study, which showed that a single dose of Pfizer’s therapy helped eliminate bleeding in 60% of patients compared to 29% in the prophylaxis arm.
Pfizer is launching a warranty program based on the durability of patient response to Beqvez treatment. This program is designed to provide greater certainty to payers and financial protection for eligible patients receiving Beqvez. The drug will be available by prescription to eligible patients this quarter. Pfizer has committed to monitor the long-term durability and safety of the treatment over 15 years.