10:25 AM EDT, 06/07/2024 (MT Newswires) -- BioMarin Pharmaceutical ( BMRN ) on Friday announced new data supporting the long-term safety and efficacy of Roctavian.

Data from study data demonstrated "durable and sustained bleed control," along with factor VIII expression, which were maintained four years post-Roctavian infusion, the company said.

Out of 134 patients receiving Roctavian, 112 had baseline annualized bleeding rate data before treatment. By year four, 73.6% had no treated bleeds, the company said, adding that over four years, the mean ABR for treated bleeds was 0.8 bleeds per year for the rollover population and 1.3 bleeds per year for all bleeds, BioMarin said.

Roctavian is a gene therapy utilizing an adeno-associated virus vector for treating severe hemophilia A in adults lacking antibodies to adeno-associated virus serotype 5, determined via a blood test, the company said.