BioMarin’s Hemophilia A Treatment Nabs Orphan Drug Status

The European Commission has granted orphan drug designation to BioMarin’s BMN 270, a gene therapy treatment for hemophilia A.

Earlier this month, the FDA awarded BMN 270 the same designation.

An AAV 5 factor VIII vector, BMN 270 is in a Phase 1/2 study evaluating the candidate in patients with severe hemophilia. The therapy is intended to restore factor VIII plasma concentrations.