Pfizer Inc. (NYSE: PFE) has announced that the U.S. Food and Drug Administration (FDA) has approved HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis. This treatment is designed to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients aged 12 and older who have hemophilia A (congenital factor VIII deficiency) or hemophilia B (congenital factor IX deficiency), both without the presence of specific inhibitors.
Pfizer has announced that HYMPAVZI™ (marstacimab-hncq) has received FDA approval as the first anti-tissue factor pathway inhibitor (anti-TFPI) for hemophilia A or B. This marks the first hemophilia treatment in the U.S. that can be administered via a pre-filled auto-injector pen. HYMPAVZI offers a subcutaneous treatment option with a once-weekly dosing schedule, requiring minimal preparation for each administration.
Dr. Suchitra S. Acharya emphasized that this approval represents a significant advancement for patients with hemophilia A or B, allowing for a manageable safety profile and simplified treatment compared to traditional intravenous infusions.
Hemophilia affects over 800,000 people worldwide, and while recent treatments have improved care, many patients still face frequent bleeding episodes. Aamir Malik from Pfizer noted that HYMPAVZI is their second hemophilia treatment approved this year, highlighting their commitment to advancing care for this community.
The approval is based on the Phase 3 BASIS trial, which showed that HYMPAVZI reduced annualized bleeding rates significantly compared to standard prophylaxis and on-demand treatments. The most common side effects reported were injection site reactions, headaches, and itching.
The treatment aims to alleviate the burden of frequent intravenous infusions, which can be time-consuming for patients. Phil Gattone from the National Bleeding Disorders Foundation praised Pfizer’s efforts in developing this innovative option.
In addition to its U.S. approval, HYMPAVZI received a positive opinion from the European Medicines Agency for similar use in adults and adolescents. Pfizer continues to expand its portfolio with new treatments for hemophilia, including gene therapies.
HYMPAVZI targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI) to help restore normal blood clotting. The safety profile remains consistent with earlier trials, and ongoing studies will further investigate its efficacy in various patient populations.
