BioMarin to present positive data on Roctavian durability at EAHAD Congress

BioMarin Pharmaceutical announced that new data on Roctavian will be presented at the 2024 European Association for Haemophilia and Allied Disorders – EAHAD – Congress, Feb. 6-9. Results from a Phase 2, open-label study of Roctavian showed that median factor VIII activity at year seven remained in the mild hemophilia range, and mean annualized bleeding rate for treated bleeds over the full follow-up period decreased by 96% from baseline for adults with severe hemophilia A in the 6×1013 vg/kg dose cohort. The majority of participants maintained hemostasis. No new safety signals emerged. The European Commission granted conditional marketing authorization to Roctavian on August 24, 2022. The FDA approved Roctavian on June 29, 2023.