BioMarin's Hemophilia A Gene Therapy Roctavian Nears Finish Line in U.S. and Europe

The long wait may be over. A one-time treatment for severe hemophilia A is now on the cusp of approval on two continents, as BioMarin Pharmaceutical announced a major regulatory update for Roctavian, its investigational gene therapy.

Navigating the FDA's High Bar

In the U.S., BioMarin has resubmitted its Biologics License Application (BLA) for Roctavian to the Food and Drug Administration (FDA). This move follows the FDA's previous Complete Response Letter (CRL), which requested two years of follow-up data from the Phase 3 GENEr8-1 study to assess the treatment's long-term durability. BioMarin stated it has now provided that data, and it anticipates a six-month review period, targeting a potential FDA decision in the second quarter of 2026.

Europe Signals a Green Light

Across the Atlantic, the path looks smoother. BioMarin reported that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending conditional marketing authorization for Roctavian. This recommendation paves the way for a final decision by the European Commission (EC) in the third quarter of 2026. If approved, Roctavian would become the first gene therapy for hemophilia A available in Europe.