FDA approves Beqvez(fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with hemophilia B.- Pfizer

Beqvez is a one-time treatment that is 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 factor. 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 utilization. According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia

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 Beqvez, and offer financial protection by insuring against the risk of efficacy failure.

Bevqez is currently under review with the European Medicines Agency (EMA), and the treatment recently received regulatory approval in Canada in January 2024.