secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2024-06-12T23:59:59+00:00
Accession:  0000950170-24-072414
Event type: regulatory
Sentiment:  positive
Materiality: 0.75
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

FDA agrees CSF heparan sulfate as surrogate endpoint for UX111 accelerated approval BLA in MPS IIIA
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- FDA agreed cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint supporting accelerated approval for UX111 gene therapy in Sanfilippo syndrome type A (MPS IIIA).
- Company intends to file BLA late 2024 or early 2025 after a pre-BLA meeting with the FDA.
- BLA filing will rely on data from the ongoing pivotal Transpher A study and long-term follow-up; results were presented at WORLDSymposium.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024072414/0000950170-24-072414-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000095017024072414/rare-20240612.htm
  HTML page:      https://secwatch.observer/filing/0000950170-24-072414

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