The European Commission (EC) has approved Pfizer's Hympavzi (marstacimab) for routine prophylaxis in hemophilia A and B patients aged 12 years and older without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). This approval marks a significant advancement in hemophilia treatment, offering a new option for patients to prevent or reduce the frequency of bleeding episodes.
Hympavzi is administered once weekly and uniquely targets tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein. By inhibiting TFPI, Hympavzi promotes blood clot formation, addressing the underlying cause of hemophilia-related bleeding.
The EC's decision is supported by data from the Phase III BASIS study, which evaluated Hympavzi over a 12-month period. The study demonstrated a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) compared to the current standard-of-care treatment. These results led to the FDA approval of Hympavzi for a similar indication last month.
Hemophilia is a rare genetic blood disorder resulting from a deficiency in clotting proteins, specifically FVIII in hemophilia A and FIX in hemophilia B. This deficiency impairs normal blood clotting, leading to potential internal bleeding, especially in joints, causing scarring and damage. The current treatment landscape includes factor replacement therapy and, more recently, gene therapies.
Pfizer continues to expand its presence in the hemophilia space. In July, the EC approved Durveqtix (fidanacogene elaparvovec), a one-time gene therapy for hemophilia B, also approved by the FDA under the name Beqvez. Additionally, Pfizer is developing giroctocogene fitelparvovec, an investigational gene therapy for hemophilia A, which showed positive results in the Phase III AFFINE study, demonstrating a statistically significant reduction in ABR compared to the pre-infusion period.
Other companies are also developing novel therapies for hemophilia. Novo Nordisk's concizumab, an anti-TFPI inhibitor, is under regulatory review for hemophilia A and B with inhibitors. BioMarin Pharmaceuticals' Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A, was approved by the FDA last year.
