BioMarin Pharmaceutical Reports Sustained Efficacy for Hemophilia A Gene Therapy in Five-Year Data

MT Newswires Live

2025/06/24

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.

Price: 54.00, Change: -0.08, Percent Change: -0.14

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