Posted by Michael Wonder on 31 Dec 2025
Ultragenyx completes rolling submission of biologics license application to US FDA for DTX401 AAV gene therapy for glycogen storage disease type Ia
30 December 2025 - If approved, DTX401 will be the first therapy approved to treat the underlying cause of glycogen storage disease type Ia.
Ultragenyx today announced that it has completed the rolling submission of its biologics license application to the US FDA seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for glycogen storage disease type Ia.
