5 Key Takeaways

• 1

  The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome type A, targeting neurological symptoms in pediatric patients.

• 2

  Fayuvi is a one-time AAV9 gene therapy that delivers a functional SGSH gene to restore enzyme production and reduce heparan sulfate accumulation.

• 3

  In a study, treated patients showed a mean 16-point increase in cognitive scores, while untreated patients experienced a 7.6-point decline.

• 4

  The most common adverse reaction to Fayuvi was elevated liver enzymes, occurring in 85% of treated patients, along with other potential risks.

• 5

  Fayuvi was originally developed at Nationwide Children’s Hospital and is now produced by Ultragenyx and Andelyn Biosciences.