Pfizer Inc. (PFE) announced on Friday that the U.S. Food and Drug Administration has granted approval for HYMPAVZI (marstacimab-hncq). This treatment serves as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in both adults and pediatric patients aged 12 and above diagnosed with hemophilia A (a congenital deficiency of factor VIII, without inhibitors) or hemophilia B (a deficiency of factor IX, without inhibitors).
The approval follows Phase 3 study results that highlighted a significant reduction in bleeding incidents compared to both routine prophylaxis and on-demand treatments in eligible patients with hemophilia A or B who do not have inhibitors.
In the United States, HYMPAVZI marks a milestone as it is the first once-weekly subcutaneous prophylactic treatment available for individuals with hemophilia B, and it is also noteworthy for being the first to allow administration through a pre-filled pen or syringe for patients with either hemophilia A or B.
Hemophilia represents a group of rare genetic blood disorders caused by deficiencies in clotting factors—specifically factor VIII in type A and factor IX in type B—affecting over 800,000 people worldwide. Diagnosed early in childhood, hemophilia hampers the blood's clotting ability, thereby raising the risk of recurring joint bleeding, which can ultimately lead to permanent joint damage.