Regulatory Approvals

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

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U.S. FDA — The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.

Read the original at U.S. FDA ↗

Auto-published by the finit.news Events monitor from U.S. FDA; summarized by AI and may contain errors — verify against the primary source. September 17, 2026.

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