- In March 2025, The U.S. FDA approved Qfitlia (fitusiran), the first and only RNA interference (RNAi) therapeutic for the treatment of hemophilia A and B, including patients with or without inhibitors. This approval marks a significant advancement in the treatment options available for individuals with these conditions
- In February 2025, Pfizer announced the cessation of global development and commercialization of its hemophilia B gene therapy, Beqvez. The decision was attributed to limited demand from patients and clinicians, reflecting broader challenges in the adoption of gene therapies within the hemophilia treatment landscape
- In December 2024, The U.S. Food and Drug Administration approved Novo Nordisk's Alhemo, a subcutaneous treatment designed to prevent or reduce bleeding episodes in hemophilia patients aged 12 years and older who have developed inhibitors against clotting factor replacements. This approval offers a new therapeutic option for patients with limited treatment choices due to the presence of inhibitors
- In June 2024, BioMarin Pharmaceutical Inc. presented new Phase 3, four-year data at the International Society on Thrombosis and Haemostasis 2024 Congress, underscoring the long-term safety and efficacy of ROCTAVIAN (valoctocogene roxaparvovec-rvox) in adults with severe hemophilia A. The data demonstrated durable and sustained bleed control and Factor VIII expression maintained four years post-infusion.
- In March 2021, CSL Behring announced a donation of 500 million international units (IUs) of coagulation factor therapy to the World Federation of Hemophilia (WFH) in an effort to promote reliable access to treatment for bleeding disorders, including hemophilia, across more than 60 developing countries. This donation is part of CSL Behring's ongoing commitment to support the WFH Humanitarian Aid Program
