BioMarin Pharmaceutical Reports Sustained Efficacy for Hemophilia A Gene Therapy in Five-Year Data
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BioMarin Pharmaceutical said Tuesday that new five-year data from its Phase 3 study showed roctavian maintained bleed control and factor VIII expression in adults with severe hemophilia A. The trial found 81.3% of participants remained off preventive treatment and 77.8% had no treated bleeds in the fifth year, the company said. No new safety signals were observed, and no participants developed inhibitors, it added.
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