The EHC acknowledges the recent decisions by several pharmaceutical companies to discontinue or refocus the development or commercialisation of haemophilia therapies since October 2024.

As most recent decision among these, at the end of February 2025 Pfizer announced that it will halt the global commercialisation of fidanacogene elaparvovec-dzkt, a gene therapy for haemophilia B, citing low demand. Approved in multiple regions, including the European Union, this one-time therapy will no longer be pursued, with Pfizer shifting focus to marstacimab-hncq, an injectable treatment for haemophilia A and B.

The EHC recognises that these decisions come at a time when significant unmet needs persist in the bleeding disorders community. While progress has been made, many people with haemophilia (PWH) or other bleeding disorders still experience treatment burdens, breakthrough bleeding, and reduced quality of life. Access to standard prophylaxis remains a challenge in many European countries, further underscoring the need for continued research, innovation, and equitable treatment availability.

The EHC urges pharmaceutical companies, health authorities, and stakeholders to remain committed to advancing treatment options for haemophilia and other bleeding disorders. Investment in research and development must continue to ensure that the needs of the bleeding disorders community are met and that all patients have access to safe, effective and innovative therapies.