The World's First Gene Therapy For Haemophilia Approved! A Single Infusion That Greatly Reduces Patient Bleeding!
The European Union has recently approved Roctavian, the first gene therapy for haemophilia A. The therapy has led to a significant reduction in the average number of annual bleeds and the average number of annual clotting factor VIII transfusions in patients with this severe bleeding disorder, and has a good safety profile.