Ultragenyx Pharmaceutical Inc. (RARE), Friday announced that it has resubmitted its Biologics License Application to the U.S. Food and Drug Administration, seeking accelerated approval for UX111 AAV9 gene therapy for the treatment of patients with Sanfilippo syndrome type A.
The company expects up to a 6-month review period from the date of resubmission per FDA regulations, with a Prescription Drug User Fee Act or PDUFA action date expected in the third quarter of 2026.
The submission includes substantial longer-term data on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data.
In the pre-market hours, RARE is trading at $23.95, down 1.30 percent on the Nasdaq.
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