Pfizer announced that its innovative hemophilia treatment, HYMPAVZI (marstacimab-hncq), has been approved by the FDA for adults and adolescents aged 12 and older with hemophilia A and B without inhibitors. HYMPAVZI, the first anti-tissue factor pathway inhibitor (anti-TFPI) therapy approved in the U.S., offers patients a once-weekly, subcutaneous injection using a pre-filled, auto-injector pen. This development marks a shift from time-intensive intravenous infusions to a more convenient, minimal-preparation option. Data from the pivotal Phase 3 BASIS study demonstrated that HYMPAVZI effectively reduced the annualized bleeding rate (ABR) by 35% to 92% in treated patients compared to other standard therapies, highlighting its potential to improve treatment adherence and reduce bleeding episodes.
Pfizer’s commitment to advancing hemophilia treatments spans over 40 years, with HYMPAVZI being its second hemophilia therapy approved in 2024. As a rebalancing agent, HYMPAVZI targets the Kunitz 2 domain of the tissue factor pathway inhibitor to restore hemostasis, thereby preventing bleeding. HYMPAVZI’s approval adds to Pfizer’s portfolio of hemophilia solutions, which also includes gene therapy and recombinant factor treatments. Pfizer, a leader in biopharmaceutical innovation, emphasizes safety and accessibility in its global healthcare offerings, aiming to set high standards for patient care and access.
