Marstacimab FDA Approval Status

Last updated by Judith Stewart, BPharm on Jan 10, 2024.

FDA Approved: No
Generic name: marstacimab
Company: Pfizer Inc.
Treatment for: Hemophilia A, Hemophilia B

Marstacimab is an anti-tissue factor pathway inhibitor (anti-TFPI) in development for the treatment of hemophilia A or hemophilia B.

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), which prevents normal blood clotting, resulting in episodes of bleeding. The most common treatment approach for hemophilia A and B is factor replacement therapy, which replaces the missing clotting factors, but approximately 25-30% of people with hemophilia A and 3-5% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII and FIX.
Marstacimab is a human monoclonal immunoglobulin G isotype, subclass 1 (IgG1) that works as a prophylactic treatment individuals with hemophilia A or hemophilia B with or without inhibitors by targeting the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that functions to prevent the formation of blood clots.
In September 2019, the U.S. FDA granted Fast Track designation to marstacimab for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A with inhibitors or hemophilia B with inhibitors.
In December 2023, the U.S. FDA has accepted the Biologics License Application (BLA) for marstacimab for individuals living with hemophilia A or hemophilia B without inhibitors to FVIII or FIX.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date in the fourth quarter of 2024.

Development timeline for marstacimab

Date	Article
Dec 11, 2023	FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B
Dec 9, 2023	Marstacimab Phase 3 Data Presented at ASH 2023 Demonstrate Significant Bleed Reduction in Hemophilia A and B
May 30, 2023	Research and Pipeline Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial
Dec 2, 2020	Pfizer Doses First Participant in Phase 3 Study Evaluating anti-TFPI Investigational Therapy, Marstacimab, for People With Severe Hemophilia A and B With or Without Inhibitors

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