Ultragenyx's FAYUVI™ Receives FDA Approval for MPS IIIA Treatment

Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has granted standard full approval for FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111. This approval is for the treatment of pediatric patients diagnosed with mucopolysaccharidosis type IIIA (MPS IIIA), also referred to as Sanfilippo syndrome Type A. FAYUVI™ marks a significant milestone as the first-ever FDA-approved treatment for this progressive and fatal neurodegenerative disease. This is Ultragenyx's second gene therapy approval, and the company has received a Priority Review Voucher as a result of this approval.