secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2026-08-19T21:24:44+00:00
Accession:  0001193125-26-357185
Event type: regulatory
Sentiment:  positive
Materiality: 0.85
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx wins FDA accelerated approval for GENGLYCOS (DTX401) in GSDIa
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- FDA granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr) in GSDIa patients aged 8+.
- Approval based on Phase 3 GlucoGene: 46 participants, cornstarch requirement reduction at Week 48 (p<0.001).
- Efficacy data from 44 mITT (DTX401 n=20, placebo n=24); crossover followed to Week 96/144.
- Post-marketing DMP: 2-year data from 50 treated and 20 control patients; 10-year total follow-up.
- Control group consists of patients with anti-AAV8 antibodies ineligible for treatment.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/0001193125-26-357185-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000119312526357185/rare-20260819.htm
  HTML page:      https://secwatch.observer/filing/0001193125-26-357185

License: Source filings: public domain (SEC EDGAR). Summaries (headline + bullets): CC-BY-4.0; attribute https://secwatch.observer