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DTX401 (pariglasgene brecaparvovec)

Ultragenyx Pharmaceutical · $RARE
Priority Review Orphan Drug BLA
PDUFA Date
August 23, 2026
Time Remaining
46 days
Review Type
Priority (6 mo)
93%
Baseline PoA
Orphan/rare disease designation approval rate
Based on FDA historical approval rates by submission type (2015–2025)
Dynamic PoA
Coming soon
xAI Sentiment

Live Company Data NMS

Updated just now · Data: FMP
Current Price
$34.88 -11.53%
$-4.55 today
Day: $34.20 – $35.77
Market Cap
N/A
52-Week Range
$18.29
$42.37
Current price is at 69% of 52-week range
⚠️ Financial data is for informational purposes only. Not investment advice. Prices may be delayed.
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Indication

Glycogen Storage Disease Type Ia (GSDIa), AAV8 gene therapy

Key Notes

FDA accepted the BLA and granted Priority Review with a PDUFA target action date of August 23, 2026. Supported by the randomized, double-blind, placebo-controlled Phase 3 GlucoGene study plus data from 52 treated patients with up to six years of follow-up. One-time AAV8 gene therapy aiming to reduce reliance on cornstarch dosing in GSDIa. Source: Ultragenyx press release (BLA acceptance, February 2026).
⚠️ Not Investment Advice: This information is provided for educational purposes only. PDUFA dates are FDA target action dates, not guaranteed approval dates. The FDA may extend the review period, issue Complete Response Letters, or take unexpected actions. Probability of approval scores are based on historical base rates and do not account for drug-specific factors. Always consult a financial advisor before making investment decisions.

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