BioMarin Pharmaceutical Inc. (BMRN) Tuesday announced new data underscoring the long-term efficacy and safety of Roctavian (valoctocogene roxaparvovec-rvox).
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.
"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.
For comments and feedback contact: editorial@rttnews.com
Business News
June 19, 2026 16:46 ET Major central banks continued to dominate the economic news flow this week too, led by the Federal Reserve, as they announced their latest policy decisions. The Federal Reserve policy session was in focus as it was the first to be led by the new chief Kevin Warsh. In Europe, central banks of the U.K. and Switzerland announced their rate decisions. In Asia, the Bank of Japan drew attention for its policy moves, while data out of China threw some light on the state of the economy.