Ultragenyx Pharmaceutical Inc, RARE, FDA Calendar, PDUFA Date

Company Name	Ultragenyx Pharmaceutical Inc RARE
Drug Name	UX111 AAV (Resubmitted BLA )
Event Name	FDA decision on UX111 AAV gene therapy for treatment of Sanfilippo syndrome type A (MPS IIIA)
Event Date	09/19/2026
Outcome Date
Outcome	pending
Drug Status	FDA declined to approve UX111 on July 11, 2025, citing specific chemistry, manufacturing, and controls (CMC) related observations that are resolvable.
Rival Drugs	If approved, UX111 would become the first-ever approved therapy for Sanfilippo Syndrome Type A in the U.S.
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