A decision is expected by June 2022. If accepted, Roctavian will be the first gene therapy approved in Europe for hemophilia A.

Is Roctavian FDA approved?

The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A.

What is Roctavian used for?

Roctavian is an investigational gene therapy that provides a functional copy of the Factor VIII gene, which is currently being evaluated in a phase III clinical trial for individuals with severe hemophilia A without inhibitors.