Statement: HFA Responds to Latest Gene Therapy Announcement

DNA strand

Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of adults with severe factor VIII deficiency, represents a significant milestone in the treatment of hemophilia A.    As a community-based, patient-centered organization, Hemophilia Federation of America (HFA) intimately understands and […]

FDA Approves BioMarin’s Gene Therapy for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. today announced the U.S. Food and Drug Administration approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is […]

Sanofi Announces Data Showing Effective Bleed Protection in Children with Severe Hemophilia A

microscope in lab

Pivotal data from the Phase 3 XTEND-Kids study evaluating ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] once-weekly prophylaxis, a factor VIII replacement therapy, in previously treated patients younger than 12 years of age with severe hemophilia A were presented recently in a late-breaking session at the Annual Meeting of the International Society on Thrombosis and […]