FDA Clears ULTRAGENYX (RARE) Gene Therapy Fayuvi for Sanfilippo Syndrome Type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with MPS IIIA, a rare inherited disorder.
The U.S. Food and Drug Administration has approved Fayuvi (rebisufligene etisparvovec-hopf), the first therapy cleared for pediatric patients with mucopolysaccharidosis type IIIA, better known as Sanfilippo syndrome type A. The agency announced the decision on Thursday. The product is a gene therapy developed by Ultragenyx Pharmaceutical (RARE).
