Pfizer’s Breakthrough Hemophilia Drug Hympavzi Wins EU Approval

EU Approval:
The European Commission has granted marketing authorization for Pfizer's Hympavzi (marstacimab) for the routine prophylaxis of bleeding episodes in adults and adolescents aged 12 years and older with severe hemophilia A or B without inhibitors to Factor VIII (FVIII) or Factor IX (FIX)[1][2].

First-in-Class Treatment:
Hympavzi is the first once-weekly subcutaneous treatment for severe hemophilia B and the first to be administered via a pre-filled auto-injector pen for both hemophilia A and B in the EU[2][3].

Clinical Efficacy:
The approval is based on positive results from the Phase 3 BASIS study, which demonstrated a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) compared to the current standard-of-care treatment for hemophilia patients[1][4].

Mechanism of Action:
Hympavzi targets tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that prevents blood clot formation, thereby enhancing coagulation in hemophilia patients[1][4].

Global Impact:
Hemophilia affects over 800,000 people worldwide, and this approval offers a significant advancement for eligible patients by providing bleed prevention with once-weekly subcutaneous administration via a pre-filled pen[2][4].

Pfizer's Hemophilia Portfolio:
This approval builds on Pfizer's commitment to improving hemophilia care, following recent approvals for its hemophilia B gene therapy, Beqvez, and ongoing development of a gene therapy for hemophilia A, giroctocogene fitelparvovec[1][4].