secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-10-07T23:59:59+00:00
Accession:  0000950170-24-113085
Event type: regulatory
Sentiment:  positive
Materiality: 0.75
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx gets FDA Breakthrough Therapy Designation for setrusumab in osteogenesis imperfecta
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- Received FDA Breakthrough Therapy Designation for setrusumab (UX143) for OI Type I, III, IV in patients ≥2 years.
- Designation based on Phase 2 data from Orbit (14-month) and Asteroid studies showing significant fracture rate reduction.
- Breakthrough Therapy aims to expedite development and review for serious diseases with preliminary clinical evidence.
- Ultragenyx collaborates with Mereo BioPharma on UX143 development.
- Designation does not guarantee approval but may speed regulatory process.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024113085/0000950170-24-113085-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024113085/rare-20241007.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-113085

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