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Pfizer Says U.S. FDA Approved Company's BEQVEZ (Fidanacogene Elaparvovec-dzkt), A One-Time Gene Therapy For Adults With Hemophilia B; Company Now Plans Launching An Innovative Warranty Program Based On Durability Of Patient Response To Treatment

Benzinga ·  Apr 26 18:48

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.

"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."

BEQVEZ is currently under review with the European Medicines Agency (EMA), and the treatment recently received regulatory approval in Canada. In addition to BEQVEZ, Pfizer currently has two other Phase 3 programs investigating gene therapy in populations where there is a high unmet need: hemophilia A (giroctocogene fitelparvovec) and Duchenne muscular dystrophy (fordadistrogene movaparvovec). Additionally, a Phase 3 trial is investigating marstacimab, a novel, investigational anti-tissue factor pathway inhibitor for the treatment of people with hemophilia A and B with and without inhibitors. A Biologics License Application and European Marketing Authorization Application for marstacimab are currently under review with the FDA and EMA, respectively.

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