FDA Accepts DTX401 Gene Therapy BLA for GSDIa with August PDUFA Date
Ultragenyx’s BLA for DTX401 AAV gene therapy in Glycogen Storage Disease Type Ia received FDA acceptance with priority review and a PDUFA action date of August 23, 2026. Phase 3 data from 52 patients over six years show reduced daily cornstarch needs, sustained euglycemia, fewer hypoglycemia episodes and improved quality of life.
1. FDA Acceptance and Priority Review
Ultragenyx’s BLA for DTX401 was accepted by the FDA and granted priority review, with a PDUFA action date set for August 23, 2026. This regulatory milestone could expedite approval of the first therapy addressing the underlying cause of Glycogen Storage Disease Type Ia.
2. Phase 3 Clinical Data Highlights
The BLA is supported by data from 52 treated patients with up to six years of follow-up. Results showed significant reductions in daily cornstarch intake, maintenance of euglycemia, lower rates of hypoglycemia and meaningful patient-reported quality-of-life improvements.
3. PDUFA Date and Next Steps
The August 23, 2026 PDUFA action date will determine approval status. Ultragenyx will engage with the FDA throughout the review process and prepare for potential commercial launch pending a favorable decision.
4. Manufacturing and Commercial Plans
If approved, DTX401 will be manufactured entirely in the U.S. at Ultragenyx’s new gene therapy facility in Bedford, Massachusetts, ensuring domestic production capacity for the rare disease market.