Beqvez FDA Approval History
Last updated by Judith Stewart, BPharm on May 21, 2024.
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.
- 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.
- FDA approval of Beqvez was based on the results from the pivotal BENEGENE-2 study, a Phase 3, open-label, single-arm study that evaluated the annualized bleeding rate (ABR) for participants treated with gene therapy versus FIX prophylaxis replacement regimen. BENEGENE-2 met its primary endpoint of non-inferiority in the ABR of total bleeds post-Beqvez infusion versus prophylaxis regimen. A mean ABR of 2.5 was observed among patients who received Beqvez in the efficacy evaluation period compared to a mean ABR of 4.5 during the lead-in pre-treatment period. Bleeds were eliminated in 60% of patients compared to 29% in the prophylaxis arm. A median ABR of zero (range of 0 to 19) was observed during the efficacy evaluation period compared to the prophylaxis arm in which a median ABR of 1.3 (range of 0 to 53.9) was observed.
- Beqvez is administered as an intravenous infusion over approximately 60 minutes.
- Warnings and precautions associated with Beqvez include hepatotoxicity, infusion reactions, and malignancy.
- Common adverse reactions include an increase in transaminases.
Development timeline for Beqvez
Further information
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