FDA approves Pfizer’s gene therapy Beqvez for adults with hemophilia B

26 April 2024
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The US Food and Drug Administration (FDA) has approved Beqvez (fidanacogene elaparvovec-dzkt), a gene therapy for hemophilia B.

Developed by US pharma giant Pfizer (NYSE: PFE), Beqvez is authorized 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 (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.

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