FDA approves first gene therapy treatment for Sanfilippo syndrome type A
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.
On December 23, 2021, the Food and Drug Administration (FDA) granted approval for FAYUVI, marking the first gene therapy treatment for Sanfilippo syndrome type A. This breakthrough occurred after years of research at the Jerry R. Mendell Center for Gene Therapy at Nationwide Children's Hospital, led by Kevin Flanigan, MD. The therapy targets mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a severe childhood disorder affecting the brain and spinal cord.
Sanfilippo type A, the most severe form of this group of four lysosomal storage diseases, results from a deficiency in an essential enzyme leading to the accumulation of complex polysaccharides within cells. The disease impacts many organs, particularly causing neuronal dysfunction and neuron loss, ultimately leading to early mortality.
Until this approval, no treatment had been available for this ultrarare condition, which affects 1 in 70,000 live births. FAYUVI, developed by Douglas McCarty, Ph.D., and Haiyan Fu, Ph.D., is an AAV9 gene-replacement therapy. It is administered intravenously to deliver the SGSH gene to cells in and outside the nervous system, offering hope to families affected by this devastating childhood disease.
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