FDA Approves BioMarin's Gene Therapy for Severe Hemophilia A
The FDA has approved Roctavian, the first gene therapy for adults with severe hemophilia A. Hemophilia A is a rare genetic bleeding disorder caused by a deficiency in clotting factor VIII. Roctavian is a one-time gene therapy administered through intravenous infusion, which increases blood levels of factor VIII to reduce the risk of uncontrolled bleeding. The safety and effectiveness of Roctavian were evaluated in a study of adult men with severe hemophilia A, and it was found to significantly reduce bleeding rates. Common side effects include mild changes in liver function, headache, nausea, and fatigue. The approval of Roctavian provides a new treatment option for patients with severe hemophilia A.
- FDA Approves First Gene Therapy for Adults with Severe Hemophilia A FDA.gov
- U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A PR Newswire
- BioMarin lands FDA nod for $2.9M hemophilia gene therapy Roctavian FiercePharma
- New Gene Therapy Could Face Uphill Battle Despite FDA Approval The Wall Street Journal
- US FDA approves BioMarin's gene therapy for hemophilia A AOL
Reading Insights
0
1
3 min
vs 4 min read
85%
730 → 110 words
Want the full story? Read the original article
Read on FDA.gov