secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2026-01-30T23:59:59+00:00
Accession:  0001193125-26-030590
Event type: regulatory
Sentiment:  positive
Materiality: 0.80
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx resubmits UX111 BLA for Sanfilippo type A to FDA; PDUFA expected Q3 2026
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- Resubmitted BLA for UX111 gene therapy for Sanfilippo type A on Jan 30, 2026.
- Submission addresses CMC observations from July 2025 CRL and includes additional year of clinical follow-up data.
- FDA previously acknowledged robust neurodevelopmental data; updated data show durable treatment effect and acceptable safety.
- PDUFA action date expected within a month; six-month review period suggests PDUFA in Q3 2026.
- If approved, UX111 would be the first approved therapy for Sanfilippo syndrome type A.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000119312526030590/0001193125-26-030590-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000119312526030590/rare-20260130.htm
  HTML page:      https://secwatch.observer/filing/0001193125-26-030590

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