FDA approves one-time hemophilia treatment

The US-based Food and Drug Administration (FDA) has approved pharmaceutical giant Pfizer's Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes and do not have neutralising antibodies.

Beqvez is a one-time treatment 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.

“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,” said Adam Cuker, M.D., M.S., director, Penn Comprehensive and Hemophilia Thrombosis Program. “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.”

Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.The standard of care for hemophilia B treatment is prophylactic infusions of FIX replacement therapy that temporarily replace or supplement low levels of blood-clotting. Despite prophylaxis and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes. The current standard of care also places strain on healthcare systems’ budgets and resource utilisation. According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B.

“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 U.S. Commercial Officer and Executive Vice President, Pfizer. “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.”

With Beqvez now approved for use, Pfizer is launching an innovative warranty program based on durability of patient response to treatment. The goal of the warranty is to provide greater certainty to payers, maximize access for eligible patients who receive the medicine, and offer financial protection by insuring against the risk of efficacy failure.

“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. “We are excited to have BEQVEZ as a promising treatment option for eligible people living with hemophilia B. We look forward to learning more and celebrating with the community and with Pfizer at our annual conference that is currently taking place.”


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