Business
BioMarin Presents Five-Year Phase 3 Results Reinforcing Long-Term Efficacy and Safety of ROCTAVIAN® (valoctocogene roxaparvovec-rvox) at International Society on Thrombosis and Haemostasis 2025 Congress
Data from longest and largest hemophilia gene therapy study demonstrates sustained factor VIII expression and bleed control, with 81.3% of individuals
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"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\nNo new safety signals observed\nSAN 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.\n\n \n \n \n \n \n \n\n \nThe 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.\n\"I am impressed that the strong efficacy and safety results for ROCTAVIAN are sustained five years after individuals with severe hemophilia A received gene therapy, with only one adult returning to prophylaxis since the last data cut,\" said Andrew Leavitt, M.D., director of the University of California San Francisco's program for noncancerous blood disorders and Adult Hemophilia Treatment Center. \"These results highlight the role that gene therapy may play in addressing the burdens of chronic disease management in people living with hemophilia A, while maintaining excellent bleed control.\"\nAmong 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.\nAfter five years, FVIII activity was nearly stable compared to year four, with mean FVIII activity in the mild hemophilia range (one-stage assay = 24.0 IU/dL; chromogenic assay = 13.7 IU/dL); 73.5% of participants had FVIII levels in the mild hemophilia to normal range. The mean ABR for treated bleeds ...