With Roctavian as a launching pad, BioMarin looks to up pace of new drug filings

This June, the FDA cleared Roctavian as a one-time gene therapy for adults with severe hemophilia A. For BioMarin, that was just the start, with plans to expand within the hemophilia population to adolescents, for instance, or into the Japanese market. The same goes for Voxzogo, which snagged FDA approval in achondroplasia—a genetic disorder that impairs bone growth and is the most common form of disproportionately short height—in late 2021.