BioMarin Reports Positive Data From Valoctocogene Roxaparvovec Study

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) said three-year data from ongoing Phase 1/2 study of Valoctocogene Roxaparvovec gene therapy confirmed a sustained reduction in bleeds and factor VIII usage in adults with severe hemophilia A. The company said the available data shows that a loss of factor VIII expression is dependent on the level of expression and appears to decline slower as time and expression level decline. The Factor VIII activity levels are approaching a plateau and project sustained Factor VIII expression for an extended period of time.

The valoctocogene roxaparvovec was granted Breakthrough Therapy Designation by the U.S. FDA.