FDA approves Pfizer’s Beqvez for haemophilia B treatment

Pfizer has announced that the US Food and Drug Administration (FDA) has approved Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate-to-severe haemophilia B.
The drug is approved for patients with haemophilia B who ‘currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening haemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralising antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test’, according to the company’s press release.
Beqvez is a one-time treatment that aims to enable patients to produce FIX themselves, compared to the current standard-of-care that includes several intravenous infusions of FIX administered regularly.
Aamir Malik, chief US commercial officer and executive vice president at Pfizer, commented: “This milestone is a testament to Pfizer’s continued effort to advance the standard of care for people living with haemophilia, 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. We are leveraging our expertise that comes with more than 40 years of experience in the haemophilia space, and are proactively working with treatment centres, payers and the haemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it.”
Kim Phelan, chief operating officer at The Coalition for Hemophilia B, added: “For people living with haemophilia, 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. We are excited to have Beqvez as a promising treatment option for eligible people living with haemophilia B. We look forward to learning more and celebrating with the community and with Pfizer at our annual conference that is currently taking place.”
Adam Cuker MD MS, director of the Penn Comprehensive and Hemophilia Thrombosis Program, stated: “Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues. A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”