FDA approves gene therapy for MPS IIIA

The one-time intravenous treatment uses adeno-associated virus serotype 9 to deliver a functional copy of the SGSH gene.

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The FDA approved rebisufligene etisparvovec-hopf (Fayuvi) as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA.
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Mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, leads to progressive brain and nervous system damage in children.
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Rebisufligene etisparvovec is administered as a single intravenous infusion, introducing a functional SGSH gene into patients' cells.
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Cognitive function in treated patients aged 2 to 5 years remained stable or improved compared to an untreated historical control cohort.
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Adverse reactions reported included increased liver enzymes, nausea, fever, and potential long-term risks such as tumor development.

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