FDA accepts Ultragenyx's BLA for DTX401 gene therapy for GSDIa with Priority Review, PDUFA Aug 23, 2026

Ultragenyx Pharmaceutical Inc.

FDA accepted Biologics License Application for DTX401 (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia.
Granted Priority Review; Prescription Drug User Fee Act (PDUFA) action date is August 23, 2026.
DTX401 is an AAV-based gene therapy targeting a rare metabolic disorder with no approved therapies.
BLA acceptance triggers regulatory milestone; company may be eligible for priority review voucher if approved.

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