Ultragenyx Pharmaceutical Says FDA Grants Standard Full Approval Of FAYUVI For Treatment Of Pediatric Patients With Mucopolysaccharidosis Type IIIA

Ultragenyx Pharmaceutical Inc. announced that the FDA has granted standard full approval for FAYUVI (rebisufligene etisparvovec-hopf), the first-ever treatment for pediatric patients with mucopolysaccharidosis type IIIA (Sanfilippo syndrome Type A). This marks Ultragenyx's second gene therapy approval and sixth overall FDA approval. The company plans to ship the commercial product to Qualified Treatment Centers within 30-60 days and received a Priority Review Voucher upon approval.