DTX401 (pariglasgene brecaparvovec)
Glycogen storage disease type Ia · Patients with glycogen storage disease type Ia
RARE · Ultragenyx Pharmaceutical
Regulatory designations
Full timeline
7 milestonesFDA decision expected
PDUFA target action date for the DTX401 BLA.
Ultragenyx — BLA acceptance and August 23 action date↗FDA did not anticipate an advisory committee meeting
Ultragenyx reported that FDA did not anticipate convening an advisory committee for the DTX401 application at that time.
Ultragenyx — First-quarter 2026 regulatory update↗FDA accepted the BLA with Priority Review
FDA accepted the application, granted Priority Review and assigned the August 23, 2026 action date.
Ultragenyx — FDA acceptance and Priority Review↗Rolling BLA submission completed
Ultragenyx completed the rolling submission seeking approval of DTX401 as a treatment for GSDIa.
Ultragenyx — DTX401 rolling BLA completed↗Related News
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
Jul 28, 2026Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
Jul 24, 2026Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Jul 8, 2026Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
Jun 19, 2026Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)