Ultragenyx secures FDA approval for first treatment of Sanfilippo syndrome type A

The US FDA granted full approval to Ultragenyx's Fayuvi, the first gene therapy for Sanfilippo syndrome type A, a rare pediatric neurodegenerative disorder, following regulatory setbacks including a prior complete response letter. The therapy is a single-dose intravenous treatment with specific monitoring requirements, and its approval signals progress in AAV gene therapy for mucopolysaccharidosis diseases.

This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 17 Sep 2026 and 28 Sep 2026.