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Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa).

articleUltragenyx Pharmaceutical Inc.February 23, 20264/company/ultragenyx/news/ultragenyx-announces-us-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-for-dtx401-aav-gene-therapy-for-glycogen-storage-disease-type-ia-gsdia
Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

About this update from Ultragenyx Pharmaceutical Inc.

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Ultragenyx Pharmaceutical Inc.Glycogen Storage DiseaseBiologics License ApplicationFood and Drug Administrationgene therapyEuropean Medicines Agencypatientsglycogen levelsthe AgencyBLAPDUFA