Beqvez FDA Approval History
FDA Approved: Yes (First approved April 26, 2024)
Brand name: Beqvez
Generic name: fidanacogene elaparvovec-dzkt
Dosage form: Injection
Company: Pfizer Inc.
Treatment for: Hemophilia B
Beqvez (fidanacogene elaparvovec-dzkt) is an adeno-associated virus vector-based gene therapy for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency).
- Beqvez is indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:
- currently use factor IX 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. - Hemophilia B is a rare genetic bleeding disorder caused by a deficiency of a specific protein in the blood called clotting factor IX. The current standard of care of hemophilia B requires recurrent intravenous infusions of either plasma-derived or recombinant factor IX to control and prevent bleeding episodes.
- Beqvez is a gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX (FIX) gene. It is a one-time treatment that enables patients with hemophilia B to produce the factor IX themselves, rather than needing the regular intravenous infusions.
- Beqvez is administered as an intravenous infusion over approximately 60 minutes.
Development timeline for Beqvez
Further information
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