STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease

STAT PlusBiotech FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease Drug is the first treatment for the condition, sometimes known as childhood Alzheimer’s Manage alerts for this article Save this story for later Share this article By Jason MastSept. 17, 2026 General Assignment Reporter Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.

The Food and Drug Administration on Thursday approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s. The therapy, called Fayuvi, was developed by Ultragenyx. It is the first drug specifically approved to treat Sanfilippo.

Ultragenyx did not immediately release a price.Advertisement “It’s hard to overstate what this approval would mean for everybody who is living with this really horrific disease and watching their children suffer and pass away early, and all the rest that it brings,” Cara O’Neill, chief science officer of the Cure Sanfilippo Foundation, said in an interview earlier this week. “This, gosh, would just finally mean when patients and families receive this shocking diagnosis, they wouldn’t be told to … take their kids home and love them. They would be given hope and an action plan for treatment.” STAT+ Exclusive Story Already have an account?

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