RARE

DTX401 (pariglasgene brecaparvovec)

FDA PDUFA Target Action Date · Ultragenyx Pharmaceutical Inc.

Sunday, August 23, 2026

Approved

approved

Note: Accelerated approval 2026-08-19 as GENGLYCOS (pariglasgene brecaparvovec-opnr) for GSDIa in patients 8 years and older. CBER product, not yet in drugs.json. Source: https://www.globenewswire.com/news-release/2026/08/19/3348019/0/en/ultragenyx-announces-u-s-fda-approval-of-genglycos-gene-therapy-the-first-ever-fda-approved-treatment-designed-to-treat-the-underlying-cause-of-glycogen-storage-disease-type-ia-gsd.html ; 8-K https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm

Drug Information

Drug Name: DTX401 (pariglasgene brecaparvovec) (CT.gov)
Application Type: BLA
Indication: glycogen storage disease type Ia (GSDIa)
Approved As: DTX401 (pariglasgene brecaparvovec)

Filing Details

Company: Ultragenyx Pharmaceutical Inc. →
Stock Ticker: RARE
SEC CIK: 0001515673
Source: SEC EDGAR (LLM-extracted from primary filing via full-text search) →

What is a PDUFA Date?

A PDUFA (Prescription Drug User Fee Act) date is the FDA's target deadline to complete review of a drug application. The FDA aims to make an approval decision by this date.

Standard Review: 10 months from filing acceptance
Priority Review: 6 months from filing acceptance
Possible Outcomes: Approval, Complete Response Letter (CRL), or extension

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Data sourced from SEC EDGAR filings. Last updated: 2026-05-10. Report an issue