FDA accepts resubmitted BLA for UX111 gene therapy; PDUFA date Sept 19, 2026

Ultragenyx Pharmaceutical Inc.

FDA accepted for review resubmitted BLA for UX111 (rebisufligene etisparvovec) AAV9 gene therapy.
UX111 is intended for Sanfilippo syndrome Type A (MPS IIIA) under accelerated approval pathway.
PDUFA action date set for September 19, 2026.
No new safety or efficacy data disclosed; review outcome remains uncertain.

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