FDA approves Fayuvi as first gene therapy for Sanfilippo syndrome type A
The FDA has approved Fayuvi as the first gene therapy for children with Sanfilippo syndrome type A, introducing the first treatment designed to address the underlying cause of the rare neurodegenerative disorder.
Fayuvi, or rebisufligene etisparvovec-hopf, is approved for paediatric patients with mucopolysaccharidosis type IIIA and delivers a functional copy of the SGSH gene to support production of the deficient enzyme.
The approval marks a major milestone for families affected by Sanfilippo syndrome type A, a progressive inherited condition that previously had no approved disease-modifying treatment.
Field | Content |
Alert Type | Approval |
Drug Name | Rebisufligene etisparvovec-hopf |
Brand Name | Fayuvi |
Company | Ultragenyx Pharmaceutical |
Regulatory Authority | U.S. Food and Drug Administration |
Approval Type | Gene therapy approval |
Indication | Paediatric patients with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A |
Therapy Area(s) | Rare disease; neurology; genetic disease |
Technology or Modality | AAV-based gene therapy |
Target | SGSH gene / heparan N-sulfatase deficiency |
Geography | United States |
What Happened | The FDA approved Fayuvi as the first gene therapy and first approved treatment for paediatric patients with Sanfilippo syndrome type A. |
Why It Matters | The approval introduces the first disease-modifying option for a progressive neurodegenerative disorder that previously had no approved treatment targeting the underlying genetic cause. |
Supporting Context | Sanfilippo syndrome type A is caused by pathogenic variants in the SGSH gene, resulting in deficiency of the heparan N-sulfatase enzyme and progressive accumulation of heparan sulfate. |
Key Takeaway | Fayuvi becomes the first approved therapy for Sanfilippo syndrome type A and a major new milestone in paediatric gene therapy. |
What to Watch | Commercial rollout, treatment-centre readiness, long-term follow-up and real-world evidence on neurological and developmental outcomes. |
Primary Source | U.S. Food and Drug Administration |
Relevant Date | 17 September 2026 |
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