The EHC released the first 2025 issue of its Novel Treatment Review (NTR), a periodic review of novel treatments in haemophilia, von Willebrand disease, 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:

• Concizumab (brand name Alhemo), an anti-tissue-factor pathway inhibitor (TFPI), received EU marketing authorisation.
• European Commission has granted marketing authorisation for marstacimab (brand name Hympavzi), intended for prophylaxis of bleeding episodes in patients aged 12 years and older, weighing at least 35 kg, who have severe haemophilia A or B.
• Sangamo Therapeutics regains development and commercialisation rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe haemophilia A, following a decision by Pfizer to terminate the global collaboration and license agreement between the parties.
• Spark Therapeutics / Roche announced the discontinuation of dirloctocogene samoparvovec (SPK 8011, adeno-associated viral vector with B-domain deleted human FVIII gene) in adults with severe or moderately severe haemophilia A.

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