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FDA, EMA to Review Hemophilia Treatment

March 6, 2024

April 2024

The U.S. Food and Drug Administration (FDA) accepted Pfizer’s Biologics License Application for marstacimab as treatment for hemophilia A or hemophilia B in patients without inhibitors to factor VIII or factor IX. The Prescription Drug User Fee Act action date is set for the fourth quarter of 2024.

Marstacimab is a human monoclonal immunoglobulin G isotype. It works by targeting the Kunitz 2 domain of tissue factor pathway inhibitor.

The treatment is being investigated as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in a phase III, open-label, multicenter trial dubbed BASIS. Pfizer is also investigating the treatment in children with hemophilia A or B with or without inhibitors in the open-label BASIS KIDS study.

“Marstacimab has demonstrated that it may be an efficacious treatment option with once-weekly, subcutaneous flat-dose administration via an auto-injector pen for appropriate patients, if approved. This is critical as intravenous infusions are typically required for people living with these diseases today,” said James Rusnak, MD, PhD, senior vice president and chief development officer of internal medicine and infectious diseases and research and development at Pfizer.

Marstacimab was previously granted Fast Track Designation by the FDA in 2019 for the treatment of hemophilia A with inhibitors or hemophilia B with inhibitors as routine prophylaxis. The medication is also being reviewed by the European Medicines Agency (EMA).

Source: Pfizer, December 11, 2023.

 

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