Ultragenyx Receives FDA Priority Review for DTX401 Gene Therapy for GSD Type Ia
The FDA has accepted Ultragenyx's BLA for DTX401, an AAV gene therapy for Glycogen Storage Disease Type Ia, granting Priority Review with a PDUFA action date of August 23, 2026.
Ultragenyx Pharmaceutical announced the U.S. Food and Drug Administration has accepted for review the Biologics License Application seeking approval of DTX401 AAV gene therapy for the treatment of Glycogen Storage Disease Type Ia. The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act action date of August 23, 2026.
Current dietary approaches to managing GSDIa place an extraordinary burden on individuals and families while still leaving patients with significant medical needs, including the risk of potentially life-threatening episodes of acute hypoglycemia and accumulation of long-term complications over their lifetime, according to the company's chief medical officer. If approved, DTX401 would be the first treatment to address the disease at its root cause.
Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.