Pfizer has received marketing authorization from the European Commission for HYMPAVZI (marstacimab), a treatment for hemophilia A and B. This approval, based on positive Phase 3 study results, marks a significant advancement in hemophilia care, offering the first once-weekly, subcutaneous treatment option for individuals with severe hemophilia B in the EU. The drug can be administered via a pre-filled, auto-injector pen, potentially reducing the treatment burden for patients.
Hemophilia, a rare genetic blood disease affecting over 800,000 people worldwide, results from a deficiency in clotting factors FVIII (hemophilia A) or FIX (hemophilia B). This deficiency impairs blood clotting, leading to an increased risk of internal bleeding, particularly in joints and organs. Current treatments often involve regular intravenous infusions of FVIII or FIX, sometimes multiple times per week.
Clinical Efficacy and Safety
The European Commission's decision was supported by data from the pivotal Phase 3 BASIS study. The trial included adults and adolescents (12 years and older) with severe hemophilia A or B without inhibitors. Results indicated that HYMPAVZI significantly lowered the number of treated bleeds, with a 35% reduction in the annual bleeding rate. According to Pfizer, this demonstrated non-inferiority and superiority compared to routine prophylaxis with FVIII or FIX administered as part of the usual care.
Addressing Treatment Burden
"There is a considerable treatment burden associated with the standard-of-care options for hemophilia A and B, including time-consuming preparation and administration of infusions and injections potentially causing missed doses and an increased risk of bleeding," said Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants Malades Hospital, Paris. "HYMPAVZI is a significant advancement for eligible patients in that it may provide bleed prevention as well as once-weekly subcutaneous administration via a pre-filled pen."
Impact on Patient Care
Alexandre de Germay, Chief International Commercial Officer and Executive Vice President of Pfizer, stated, "HYMPAVZI offers a first-in-class treatment option for people living with hemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities as simple as climbing stairs. This approval builds on Pfizer’s more than four-decade commitment to improve the standard of care in hemophilia, and we look forward to delivering this medicine that reduced bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients."
