Pfizer Announces FDA and EMA Acceptance of Marstacimab BLA/MAA for Hemophilia A and B

China Finance Network December 14th: Pfizer, Inc. recently announced that the Biologics License Application (BLA) for marstacimab, a novel anti-Tissue Factor Pathway Inhibitor (anti-TFPI), used to treat patients with Hemophilia A and Hemophilia B without inhibitors in their bodies, has been accepted by the U.S. Food and Drug Administration (FDA). The Marketing Authorization Application (MAA) for marstacimab in Europe has also passed the acceptance verification and is currently under review by the European Medicines Agency (EMA).

The FDA is planning to make a decision on the review of the Prescription Drug User Fee Act (PDUFA) in the fourth quarter of 2024, and the European Commission is expected to make a decision in the first quarter of 2025. If marstacimab is approved in the United States and the European Union, it is expected to become the first once-weekly subcutaneous injection for the treatment of hemophilia B, as well as the first fixed-dose medication for the treatment of both hemophilia A and hemophilia B.

James Rusnak, MD, PhD, Senior Vice President of Pfizer Global Product Development and Chief Development Officer of Internal Medicine and Hospital Care, stated that Marstacimab is a once-weekly, fixed-dose medication administered subcutaneously via an auto-injector pen. If approved, it will provide an effective treatment option for hemophilia. "At this stage, patients suffering from hemophilia typically require intravenous infusion therapies. We look forward to collaborating with the FDA, EMA, and more global regulatory agencies to advance the review of this novel therapeutic, ensuring this important product benefits patients worldwide as soon as possible."

For over fifty years, replacement therapy with clotting factors has been the conventional treatment for hemophilia A and hemophilia B, promoting normal blood coagulation by supplementing the missing clotting factors. Marstacimab is a novel hemophilia treatment that improves coagulation function by inhibiting the TFPI pathway.

The regulatory application for Marstacimab is based on the efficacy and safety data obtained from the Phase III BASIS study (NCT03938792). The key results of this study were presented during the American Society of Hematology (ASH) Annual Meeting held on December 9, 2023. Compared with routine prophylactic treatment and on-demand treatment, marstacimab reduced the annualized bleeding rate by 35% in hemophilia A patients and by 92% in hemophilia B patients without inhibitors. At the sixth China International Import Expo (CIIE) held in November, Pfizer Rare Disease introduced this innovative hemophilia drug and revealed that China would submit the marketing registration application in synchronization with the rest of the world, striving to be one of the first countries to launch the product. Currently, the BASIS study is enrolling patients with inhibitors, and the results are expected to be announced as early as the end of 2024. In addition to the BASIS study, Pfizer is also conducting research on two gene therapies, fidanacogene elaparvovec and giroctocogene fitelparvovec, for the treatment of adult patients with hemophilia B and hemophilia A, respectively.