$2.9 Million Gene Therapy for Severe Hemophilia Is Approved by FDA

June 30, 2023

(Associated Press) – U.S. officials on Thursday approved drugmaker BioMarin’s gene therapy for the most common form of hemophilia, a $2.9 million infused treatment that can significantly reduce dangerous bleeding problems. The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. It’s the first gene therapy for those patients. (Read More)