BioMarin Presents Long-term Efficacy And Safety Of Roctavian

(RTTNews) - 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.