- In March 2025, the U.S. FDA approved Sanofi's Qfitlia (fitusiran), a novel subcutaneous therapy for hemophilia A and B patients aged 12 and older, with or without inhibitors. Administered every two months, Qfitlia demonstrated a 90% reduction in annualized bleeding rates in clinical trials. Priced at USD 642,000 annually, it offers a significant improvement in treatment convenience and quality of life for patients
- In February 2025, Pfizer announced the discontinuation of its hemophilia B gene therapy, fidanacogene elaparvovec (marketed as Beqvez in the U.S. and Durveqtix in the EU), citing limited patient and clinician interest. Despite initial approvals, including a conditional marketing authorization from the European Commission in July 2024, the therapy has been withdrawn from the market
- In February 2025, Pfizer announced the discontinuation of its hemophilia B gene therapy, fidanacogene elaparvovec (Beqvez), citing limited patient and clinician interest despite its FDA approval in April 2024
- In January 2025, CSL Behring announced a strategic shift in its gene therapy pipeline, deprioritizing ex vivo lentiviral-based gene therapies. This decision included the closure of its R&D facility in Pasadena, California, which was previously involved in the development of Hemgenix
- In May 2024, Be Biopharma's BE-101, an engineered B Cell medicine for Hemophilia B, received Orphan Drug Designation from the U.S. FDA. The company initiated the Phase 1/2 BeCoMe-9 clinical trial in late 2024 to evaluate BE-101 in adults with severe or moderately severe Hemophilia
