Catalyst overview
Research summary
This catalyst tracks a Approved update for DTX401 in Glycogen Storage Disease Type Ia . The expected timing is based on the latest available company guidance and may change.
05/2023- Dosing complete. 5/30/24- Positive Ph3 topline results. 5/30/24- Present full 48wk data at a scientific conference later in 2024. 2/13/25- BLA submission in mid-2025. 8/18/25- Rolling BLA initiated, complete submission in Q4 2025. 9/8/25- Announces positive longer-term data. 11/04/25:- Rolling BLA submission ongoing for DTX401; expect completion in Q4 2025 including long-term data. 12/30/25:- Completed rolling BLA submission for DTX401 in GSDIa; next catalyst FDA acceptance for review. 01/12/26:- DTX401 rolling BLA completed Dec 2025; PDUFA approval decision expected in Q3 2026. 02/23/26:- FDA accepted DTX401 BLA with Priority Review; PDUFA action date set for August 23, 2026. 05/05/26:- FDA does not anticipate advisory committee meeting before DTX401 August 23, 2026 PDUFA decision. 08/04/26:- DTX401 PDUFA action date reiterated for August 23, 2026. 08/19/26:- FDA granted accelerated approval for GENGLYCOS (DTX401) in GSDIa.
Verification
Confirm timing and program details against the company announcement or filing.