Ultragenyx wins FDA approval for its Sanfilippo gene therapy

Ultragenyx ($RARE) received standard FDA approval for FAYUVI, the first approved treatment for Sanfilippo syndrome Type A. The single-infusion gene therapy treats neurological manifestations in pediatric patients with preserved neurodevelopmental function. The company expects commercial supplies to reach qualified US treatment centers within 30–60 days and also received a priority review voucher.

The supporting program compared 17 treated patients with 27 patients from an external natural-history cohort, rather than a randomized placebo group. Treated patients showed a 23.5-point advantage on the specified cognitive assessment. The label includes precautions covering liver toxicity, reduced platelets and infusion reactions, with monitoring required. Approval establishes a commercial pathway, but does not mean every affected child is eligible or that treatment reverses all existing damage.

Sources:

• Ultragenyx — approval announcement and prescribing-information summary, GlobeNewswire reprint