, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: ) today announced that new data supporting the long-term safety and efficacy of ROCTAVIAN (valoctocogene roxaparvovec-rvox) will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in , . "We are pleased to share data at ISTH demonstrating that ROCTAVIAN continues to offer durable and sustained bleed control and endogenous factor VIII expression four years after the infusion, representing the longest and largest Phase 3 follow-up results of a gene therapy in hemophilia," said , M.
D., president of Worldwide Research and Development at BioMarin. "Importantly, these Phase 3 data also indicate a plateauing of factor VIII levels after year three with the majority of patients remaining off prophylaxis, which shows ROCTAVIAN can offer long-term bleed protection for adults with severe hemophilia A and may provide relief from the burden of chronic infusions and injections.
" The Phase 3 GENEr8-1 trial demonstrated that durable bleed control and sustained factor VIII (FVIII) expression were maintained four years after treatment with ROCTAVIAN, with FVIII activity near stable compared with results reported previously and no new safety signals observed. Of the 134 patients who received ROCTAVIAN in the study, the rollover population of 112 patients had baseline annualized bleeding rate (ABR) data prospectively collected during a period of at least six months while on routine FVIII proph.