FDA approves first gene therapy for Sanfilippo syndrome type A
The FDA approved Fayuvi, a gene‑therapy for Sanfilippo syndrome type A, on Thursday. Ultragenyx is the developer, according to STAT. The therapy is the first drug cleared for the ultra‑rare condition, also known as childhood Alzheimer's. Ultragenyx has not released a price. Cara O’Neill, chief science officer of the Cure Sanfilippo Foundation, said families receiving a diagnosis will now have a treatment plan and a source of hope.
Children with Sanfilippo syndrome type A now have an FDA‑approved therapy, moving them beyond supportive care alone.
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