The EHC released the second 2025 issue of its Novel Treatment Review (NTR), a periodic review of novel treatments in haemophilia, von Willebrand Disease (VWD), and other rare bleeding disorders.

The purpose of the NTR is to provide up-to-date information to our broader community, particularly to EHC National Member Organisations (NMOs), and a general overview of the rapidly evolving landscape of coagulation product developments in rare bleeding disorders.

The key highlights in this latest edition include:

• The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Pfizer’s marstacimab, marketed as Hympavzi, for the prevention and reduction of bleeding in patients aged 12 and older with severe hemophilia A or B who have not developed inhibitors to factor VIII or IX.
• The U.S. Food and Drug Administration (FDA) has approved fitusiran, marketed as Qfitlia, as a routine prophylactic treatment to prevent or reduce bleeding episodes in people aged 12 and older with haemophilia A or B, with or without inhibitors or antibodies that can reduce the effectiveness of treatment.
• Metagenomi is conducting preclinical studies to advance MGX-001, its experimental gene-editing therapy for haemophilia A, with the goal of entering clinical trials in 2026.
• Pfizer is discontinuing the global development and commercialisation of Beqvez, its gene therapy for haemophilia B. The company will instead focus on advancing Hympavzi, an anti–tissue factor pathway inhibitor for both haemophilia A and B.

The EHC wishes to extend its gratitude to the EHC NTR Working Group for their contribution to the development of this issue.