Ultragenyx Pharmaceutical Rises After FDA Approves Fayuvi for Sanfilippo Syndrome Type A — Update
By Josh Beckerman
Shares of Ultragenyx Pharmaceutical climbed after the Food and Drug Administration approved the company's Fayuvi for children with Sanfilippo syndrome type A.
The stock closed up 13% at $14.50 Thursday but has fallen 37% for the year as of the market close. Shares were recently at $14.78 after hours.
The agency said this is the first approved treatment for Sanfilippo syndrome type A, a rare inherited disease that damages the brain and nervous system. "Until today, treatment was limited to managing symptoms; there was no FDA-approved therapy designed to change the underlying course of the disease," the FDA said.
Ultragenyx said commercial product is expected to be available to ship to qualified treatment centers within 30 to 60 days. This marks the company's second gene therapy approval and its sixth overall approval.
The company previously said there was a Sept. 19 Prescription Drug User Fee Act action date for the drug, which was then known as UX111.
Ultragenyx received a priority review voucher upon the approval.
Shares fell sharply earlier this month after the company reported unfavorable Phase 3 results for apazunersen (GTX-102) in Angelman syndrome.
Write to Josh Beckerman at josh.beckerman@wsj.com
(END) Dow Jones Newswires
September 17, 2026 17:22 ET (21:22 GMT)
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