BioMarin Pharmaceutical (BMRN) 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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