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FDA grants full approval for FAYUVI to treat pediatric patients with MPS IIIA
RAREUltragenyx Pharmaceutical Inc.
On September 17, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration granted standard full approval for FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, to treat pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA). This marks the first FDA-approved treatment for Sanfilippo syndrome Type A and serves as the company's second gene therapy approval. Additionally, Ultragenyx received a Priority Review Voucher associated with this regulatory milestone. The company operates in the biopharmaceutical industry, focusing on developing treatments for rare genetic diseases.