www.fdanews.com/articles/180433-ema-grants-biomarin-priority-medicine-support-for-hemophilia-therapy
EMA Grants BioMarin Priority Medicine Support for Hemophilia Therapy
February 10, 2017
BioMarin received a priority medicine designation from the European Medicines Agency for its gene therapy treatment for hemophilia A.
The therapy, BMN 270, is designed to alter a genetic problem by adding a corrected copy of the defective gene. In this case, it would be factor VIII, a clotting protein.