FDA Approves ULTRAGENYX (RARE) Gene Therapy for Sanfilippo Syndrome Type A
Ultragenyx wins full FDA approval for FAYUVI, the first treatment ever cleared for MPS IIIA, and receives a Priority Review Voucher.
Ultragenyx Pharmaceutical (RARE) said the U.S. Food and Drug Administration granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), previously known as UX111, for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome Type A. It is the first treatment ever approved for the disease and the second gene therapy approval for the company, its sixth FDA approval overall. Ultragenyx also received a Priority Review Voucher upon approval.
