secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2025-08-18T23:59:59+00:00
Accession:  0000950170-25-109570
Event type: other_material
Sentiment:  positive
Materiality: 0.70
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx starts rolling BLA submission for DTX401 gene therapy in GSDIa
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- Initiated rolling BLA submission to FDA for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia.
- Submitted non-clinical and clinical modules; plans to complete full BLA including CMC module in Q4 2025.
- BLA includes 96-week Phase 3 data showing ~60-64% reduction in daily cornstarch use from baseline.
- Updates address FDA observations from prior UX111 complete response letter related to CMC and manufacturing.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017025109570/0000950170-25-109570-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017025109570/rare-20250818.htm
  HTML page:      https://secwatch.observer/filing/0000950170-25-109570

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