Data from longest and largest hemophilia gene therapy study demonstrates sustained factor VIII expression and bleed control, with 81.3% of individuals remaining off prophylaxis

No new safety signals observed

SAN RAFAEL, Calif., June 24, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced new data underscoring the long-term efficacy and safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) were presented at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, D.C., June 21-25, 2025.

The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained five years after treatment with ROCTAVIAN. FVIII activity remained consistent with previously reported results, and no new safety signals were observed. Across the entire trial, no participants developed FVIII inhibitors or experienced thromboembolic events, and there were no treatment-related malignancies across the five years of the study.