Ultragenyx resubmits UX111 BLA for Sanfilippo type A to FDA; PDUFA expected Q3 2026

Ultragenyx Pharmaceutical Inc.

Resubmitted BLA for UX111 gene therapy for Sanfilippo type A on Jan 30, 2026.
Submission addresses CMC observations from July 2025 CRL and includes additional year of clinical follow-up data.
FDA previously acknowledged robust neurodevelopmental data; updated data show durable treatment effect and acceptable safety.
PDUFA action date expected within a month; six-month review period suggests PDUFA in Q3 2026.
If approved, UX111 would be the first approved therapy for Sanfilippo syndrome type A.

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