Ultragenyx's DTX401 Gene Therapy Receives FDA Priority Review
Ultragenyx Pharmaceutical announced the U.S. Food and Drug Administration, FDA, has accepted for review the Biologics License Application, BLA, 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, PDUFA, 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," said Eric Crombez, M.D., chief medical officer at Ultragenyx. "If approved, DTX401 would be the first treatment to address the disease at its root cause. We appreciate the FDA's timely acceptance of the BLA and will continue to work with the Agency throughout its review process."
