Posted by Michael Wonder on 19 Aug 2025
Ultragenyx initiates rolling submission of niologics license application to US FDA for DTX401 AAV gene therapy for the Treatment of glycogen storage disease type Ia
18 August 2025 - Ultragenyx today announced the initiation of a rolling submission of a niologics license application to the US FDA seeking approval for DTX401 AAV gene therapy as a treatment for glycogen storage disease type Ia.
The company has submitted the non-clinical and clinical modules to the FDA and plans to complete the full BLA including submission of the chemistry, manufacturing and controls module in the fourth quarter of 2025.
