
FDA Approves Pfizer’s Gene Therapy for Adults with Hemophilia B
Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy, helps adults with hemophilia B produce factor IX themselves instead of receiving regular intravenous infusions.
The FDA has
Beqvez, an adeno-associated virus-based
The approval was based on results from the Phase 3 open-label, single-arm study, BENEGENE-2 (
In people with hemophilia B, a deficiency in FIX prevents blood clotting and causes those with the genetic disorder to bleed more often and longer than others. Approximately 38,000 people worldwide live with the disorder (1). People with hemophilia B are at risk of spontaneous bleeding, even with preventive treatment and the standard of care, which includes regular infusions.
“This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with hemophilia, with the delivery of a medicine that has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration,” said Aamir Malik, chief US commercial officer and executive vice president, Pfizer, in a press release (1). “We are leveraging our expertise that comes with more than 40 years of experience in the hemophilia space, and are proactively working with treatment centers, payers, and the hemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it.”
“For people living with hemophilia, disease management can interfere with many aspects of their lives. A one-time infusion of Beqvez may allow eligible patients more time for the things they love,” said Kim Phelan, chief operating officer, The Coalition for Hemophilia B (1). “We are excited to have Beqvez as a promising treatment option for eligible people living with hemophilia B."
The first FDA-approved gene therapy for Pfizer, Beqvez comes to market on the heels of its competitor
The European Medicines Agency is also reviewing Beqvez for possible approval in Europe. Regulatory
References
- Pfizer. US FDA Approves Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B. Press Release. April 26, 2024.
https://www.pfizer.com/news/press-release/press-release-detail/us-fda-approves-pfizers-beqveztm-fidanacogene-elaparvovec - uniQure. uniQure announces FDA approval of first gene therapy for adults with hemophilia B. Press release. November 22, 2022. https://www.globenewswire.com/news-release/2022/11/22/2561267/0/en/uniQure-announces-FDA-approval-of-first-gene-therapy-for-adults-with-hemophilia-B.html
- Pfizer Canada. Health Canada Approves Pfizer Canada’s Gene Therapy in Hemophilia B. Press Release. Jan. 3, 2024.
https://www.pfizer.ca/en/media-centre/health-canada-approves-pfizer-canadas-gene-therapy-in-hemophilia-b
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