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 haemophilia B and the first treatment administered as a flat dose for people living with haemophilia A or B," the Company stated in a release.
“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.”
For more than five decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces missing clotting factors to facilitate proper blood coagulation. Marstacimab is a novel, investigational treatment for hemophilia that is designed to restore hemostasis by inhibiting TFPI. For appropriate patients living with hemophilia A and B, the goal of this treatment is to prevent potentially life-threatening bleeds with a once-weekly, subcutaneous flat-dose administration.
The submissions for marstacimab are based on efficacy and safety data from the Phase 3 BASIS trial (NCT03938792). Key findings were recently presented at the American Society of Hematology (ASH) Annual Meeting and Exposition on December 9, 2023. The inhibitor cohort of the BASIS trial has completed enrollment and is expected to read out as early as late 2024.
Read also: Pfizer Elrexfio gets European Commission approval for Multiple Myeloma
Disclaimer: This website is primarily for healthcare professionals. The content here does not replace medical advice and should not be used as medical, diagnostic, endorsement, treatment, or prescription advice. Medical science evolves rapidly, and we strive to keep our information current. If you find any discrepancies, please contact us at corrections@medicaldialogues.in. Read our Correction Policy here. Nothing here should be used as a substitute for medical advice, diagnosis, or treatment. We do not endorse any healthcare advice that contradicts a physician's guidance. Use of this site is subject to our Terms of Use, Privacy Policy, and Advertisement Policy. For more details, read our Full Disclaimer here.
NOTE: Join us in combating medical misinformation. If you encounter a questionable health, medical, or medical education claim, email us at factcheck@medicaldialogues.in for evaluation.