The FDA recently approved yet another gene therapy, Beqvez (fidanacogene elaparvovec-dzkt) from Pfizer, Inc., for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (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 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 factor IX themselves. The current standard of care requires regular intravenous infusions of factor IX typically administered multiple times a week or month.
A one-time dose of Beqvez has reduced bleeds post-treatment compared to standard of care with a median of zero bleeds (range 0 to 19) after up to three years of follow-up, providing sustained bleed protection and potentially avoiding years of treatment burden with prophylaxis for many patients.
Beqvez follows on the heels of the approval of another gene therapy for hemophilia B, Hemgenix, approved in 2023 . According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B. By comparison, upwards of 20,000 males in the US have hemophilia-A.
Pfizer set the treatment’s list price at $3.5 million. That matches the cost of Hemgenix, the other available gene therapy for hemophilia B. Pfizer will offer insurers a warranty providing “financial protections” if Beqvez doesn’t work or its effects don’t last.
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Pfizer did not announce details for logistics and distribution.
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