BioMarin sees long-term strength in Roctavian data
US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) has presented new five-year data from its pivotal Phase III trial of Roctavian (valoctocogene roxaparvovec), showing sustained bleed control and a stable safety profile for adults with severe hemophilia A.
The results, unveiled at the ISTH 2025 congress in Washington, DC, came from the GENEr8-1 study and showed that the majority of participants continued to avoid routine prophylaxis and maintained low bleed rates half a decade after a one-time infusion.
Mean factor VIII activity remained within the mild hemophilia range, with no new safety concerns emerging. Notably, 78% of participants had no treated bleeds in the fifth year of follow-up, and 81% remained off preventive treatment.
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