Pfizer’s Marstacimab Submitted for Regulatory Approval to Treat Hemophilia

New York, NY – The novel hemophilia therapeutic marstacimab has been accepted for submission by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for regulatory approval, according to a news release.

Pfizer announced in a news release that the Biologics License Application (BLA) for its antitissue pathway inhibitor candidate, marstacimab, has been accepted by the FDA for use in patients with hemophilia A or B. This therapeutic was specifically designed for patients with either hemophilic condition who do not have inhibitors for factor VIII (for hemophilia A) or factor IX (for hemophilia B).

If marstacimab is approved in the United States and the EU, it will become the first treatment that can be administered once weekly as a subcutaneous injection. As for patients with hemophilia A, this treatment will be the first that can be administered at a flat dose.

Marstacimab is uniquely welcome in the hemophilia space because it does not require intravenous infusions of clotting factors—the standard of care in hemophilia today. Factor replacement therapy is deeply entrenched in hemophilia care; it is a therapeutic strategy that has been in use for more than 5 decades and has saved countless lives. Nevertheless, the treatment burden of this strategy remains high.

The submission of marstacimab for regulatory approval is based on results from the phase 3 BASIS trial, which confirmed its efficacy and safety. Results of this study were recently presented at the American Society of Hematology Annual Meeting & Exposition.

Marstacimab is a human monoclonal immunoglobulin G isotype, subclass 1. It works by targeting the Kunitz 2 domain of the tissue factor pathway inhibitor, thereby dismantling its ability to prevent blood clot formation. Marstacimab was developed to be a prophylactic therapy intended to reduce or altogether prevent bleeding episodes in individuals with hemophilia A or B.

“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,” James Rusnak, MD, PhD, senior vice president at Pfizer, said.



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