DTX401 (pariglasgene brecaparvovec)
Glycogen storage disease type Ia · Patients with glycogen storage disease type Ia
RARE · Ultragenyx Pharmaceutical
监管认定
完整时间线
7 个里程碑FDA 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↗相关新闻
Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update
2026年7月28日Ultragenyx to Host Conference Call for Second Quarter 2026 Financial Results and Corporate Update
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2026年7月8日Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
2026年6月19日Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)