FDA and EMA accept Marstacimab regulatory submissions for the treatment of Hemophilia A and B

Submissions based on positive data from the Phase 3 BASIS trial, which were presented this past weekend at the American Society of Hematology (ASH) Annual Meeting

Pfizer announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for its anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab for individuals living with hemophilia A or hemophilia B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date in the fourth quarter of 2024, and a decision from the European Commission is anticipated by the first quarter of 2025. If approved in the U.S. and EU, marstacimab is expected to become the first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B.

“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, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Infectious Diseases, Research and Development, Pfizer.

“We look forward to progressing the review of this novel therapy with the FDA, EMA, and global regulatory authorities to bring this important medicine to patients globally.”