BioMarin reports new data underscoring the long-term efficacy of ROCTAVIAN

BioMarin (BMRN) Pharmaceutical announced new data underscoring the long-term efficacy and safety of ROCTAVIAN were presented at the 33rd Congress of the International Society on Thrombosis and Haemostasis in Washington, D.C., June 21-25, 2025. The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII 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. Among the 134 individuals who received ROCTAVIAN in the study, the rollover population of 112 patients from the 270-902 noninterventional lead-in study had baseline annualized bleeding rate (ABR) data prospectively collected during a period of at least six months while on routine FVIII prophylaxis prior to receiving ROCTAVIAN, and two of the 112 patients discontinued the study prior to year five. After five years, FVIII activity was nearly stable compared to year four, with mean FVIII activity in the mild hemophilia range; 73.5% of participants had FVIII levels in the mild hemophilia to normal range. The mean ABR for treated bleeds for the rollover population was 0.6 bleeds/year after year five. During year five, 77.8% of the remaining participants in the rollover population had zero treated bleeds. At the end of year five, 81.3% of participants remained off prophylaxis. Additionally, clinically relevant changes in health-related quality of life measures were observed over five years.