- In April 2023, CSL Behring announced positive topline results from its Phase III AFFINITY study evaluating efanesoctocog alfa, a novel recombinant factor VIII therapy for hemophilia A. The trial demonstrated extended half-life and superior bleed protection compared to existing factor VIII products. This advancement underscores CSL’s commitment to innovating long-acting therapies that enhance patient quality of life and treatment adherence while reducing infusion frequency
- In March 2023, BioMarin Pharmaceutical Inc. received European Commission approval for Roctavian (valoctocogene roxaparvovec), the first gene therapy for severe hemophilia A. The single-dose therapy offers long-term bleeding control by enabling patients to produce factor VIII endogenously. This historic approval marks a major milestone in hemophilia care and positions BioMarin as a leader in curative gene-based solutions
- In February 2023, Pfizer Inc. and Sangamo Therapeutics reported updated results from their Phase I/II Alta study of giroctocogene fitelparvovec, an investigational gene therapy for hemophilia A. The data indicated sustained therapeutic levels of factor VIII and a significant reduction in bleeding episodes over a multi-year follow-up. This collaboration reflects the growing momentum behind gene therapy as a transformative treatment pathway for hemophilia
- In January 2023, Roche’s Hemlibra (emicizumab) was included in China’s National Reimbursement Drug List (NRDL), significantly increasing its accessibility to hemophilia A patients with and without inhibitors. This development represents a critical step toward equitable treatment in emerging markets and reinforces Roche’s strategic focus on global expansion of its innovative bispecific antibody therapy
- In January 2023, Sanofi and Sobi announced a joint initiative to expand access to Alprolix and Eloctate, their extended half-life factor IX and factor VIII therapies, in underserved regions through a partnership with the World Federation of Hemophilia (WFH). This initiative aims to close treatment gaps by enhancing global distribution, education, and support infrastructure—reinforcing the companies’ commitment to equitable healthcare delivery for hemophilia patients worldwide
