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 will support access through its UltraCare® program, with commercial product expected to ship to Qualified Treatment Centers within 30-60 days. Additionally, Ultragenyx received a Priority Review Voucher upon this approval.