BioMarin (BMRN) Hemophilia Gene Therapy Gets FDA's RMAT Tag

BioMarin Pharmaceutical Inc.’s BMRN gene therapy for severe hemophilia A, valoctocogene roxaparvovec, has been given Regenerative Medicine Advanced Therapy (“RMAT”) designation by the FDA.

The RMAT designation was created under the 21st Century Cures Act and is granted to speed up the development and review of regenerative therapies that target serious or life-threatening conditions.