Positive CHMP opinion for treatment by marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents 12 years and older with severe hemophilia A and for adults and adolescents with hemophilia A and B
Pfizer Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents 12 years and older with severe hemophilia A (congenital factor VIII [FVIII] deficiency, FVIII less than 1%) without FVIII inhibitors, or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX less than 1%) without FIX inhibitors.
Marstacimab is a once-weekly treatment that, if approved, will be administered subcutaneously as a flat (not weight-based) dose with a pre-filled, auto-injector pen. If approved by the European Commission, marstacimab is expected to become the first anti-tissue factor pathway inhibitor (anti-TFPI) to receive regulatory approval for the treatment of hemophilia A and B in eligible patients. The anti-TFPI mechanism is intentionally designed with the goal of restoring balance between bleeding tendency and blood clot formation.
The Marketing Authorization Application for marstacimab is based on results from the pivotal Phase III BASIS trial (NCT03938792) to evaluate the efficacy and safety of marstacimab in adults and adolescents 12 years and older with severe hemophilia A or B without inhibitors. The inhibitor cohort of the BASIS trial is ongoing, with results expected in the third quarter of 2025.
Marstacimab is also currently under review with the FDA , with a Prescription Drug User Fee Act (PDUFA) action date set for the fourth quarter of this year.
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