Posted by Michael Wonder on 24 Feb 2026
Ultragenyx announces US FDA acceptance and priority review of the biologics license application for DTX401 AAV gene therapy for glycogen storage disease type Ia
23 February 2026 - Ultragenyx today announced the US FDA has accepted for review the biologics license application seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of glycogen storage disease type Ia.
The FDA granted the BLA priority review and assigned a PDUFA action date of 23 August 2026.
