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FDA approves first gene therapy treatment for Sanfilippo syndrome type A

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FDA approves first gene therapy treatment for Sanfilippo syndrome type A

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The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, following years of research in its Jerry R. Mendell Center for Gene Therapy to help patients with devastating diseases.

The FDA approved the treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), known as Sanfilippo syndrome type A. Kevin Flanigan, MD, director of the Jerry R. Mendell Center for Gene Therapy at the Abigail Wexner Research Institute, led the first clinical trial for a systemic gene therapy for this disorder.

"The road to approval has been long, as we dosed the first study participant in 2016," Flanigan said. "This approval is particularly gratifying because we are finally able to provide a meaningful gene therapy to families affected by such a devastating childhood disease."

Nationwide Children's will be a qualified treatment center for FAYUVI, becoming part of a network across the country with the advanced facilities and specially trained staff needed for the administration of FAYUVI.

Affecting the brain and spinal cord, Sanfilippo type A is the most severe form of Sanfilippo syndrome, a group of four devastating lysosomal storage diseases, each of which is caused by a deficiency in an essential enzyme that leads to an accumulation of complex polysaccharides within cells.

In MPS IIIA, mutations in the SGSH gene affect many organs but particularly lead to neuronal dysfunction and neuron loss.


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