Pfizer hemophilia drug marstacimab accepted for FDA, EU review

U.S. and European regulators have accepted applications for Pfizer’s marstacimab, an investigational treatment for hemophilia A and B. The FDA set a decision date in late 2024 while the EMA set the stage for a possible approval in early 2025.

The applications are based on positive data from the phase 3 BASIS trial, presented over the weekend at the American Society of Hematology’s annual conference. The data showed marstacimab reduced bleeding rates compared to standard treatments. The study, which investigated 116 people with hemophilia over a 12-month active treatment period, found that marstacimab significantly reduced bleeding rates compared to standard treatments, which include routine prophylaxis or on-demand (OD) treatment. Routine prophylaxis involves regular, scheduled doses of clotting factor concentrates to prevent bleeding episodes in individuals with hemophilia while OD treats bleeding e…

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