FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

Fayuvi is authorized for pediatric patients with preserved neurodevelopmental function following a study comparing treated children with a natural history cohort

September 30, 2026
3 min

The Medicine Maker

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The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome type A, targeting neurological symptoms in pediatric patients.
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Fayuvi is a one-time AAV9 gene therapy that delivers a functional SGSH gene to restore enzyme production and reduce heparan sulfate accumulation.
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In a study, treated patients showed a mean 16-point increase in cognitive scores, while untreated patients experienced a 7.6-point decline.
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The most common adverse reaction to Fayuvi was elevated liver enzymes, occurring in 85% of treated patients, along with other potential risks.
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Fayuvi was originally developed at Nationwide Children’s Hospital and is now produced by Ultragenyx and Andelyn Biosciences.

FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

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Original Source(s)

FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A

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