FDA Approves BioMarin's Gene Therapy for Hemophilia A

The FDA has approved Roctavian, a gene therapy treatment for severe hemophilia A, a rare and potentially fatal blood disorder. The treatment involves a single infusion that inserts missing genes and could eliminate the need for frequent injections. Roctavian is the latest in a series of FDA-approved gene therapies, signaling the potential for gene therapy to revolutionize the treatment of various diseases. However, the high cost of gene therapies remains a significant barrier to widespread use.