DTX401 (pariglasgene brecaparvovec) is an investigational AAV8 gene therapy candidate for glycogen storage disease type Ia (GSDIa). It is designed as a one-time intravenous treatment to restore G6Pase expression and address disease biology at the liver level. The program is under FDA Priority Review with a PDUFA action date in August 2026.
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 confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.
