secwatch.observer — SEC 8-K summary
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Issuer:     Ultragenyx Pharmaceutical Inc. (RARE)
CIK:        0001515673
Form:       8-K
Filed at:   2026-09-17T19:55:37+00:00
Accession:  0001515673-26-000006
Event type: regulatory
Sentiment:  positive
Materiality: 0.80
Item codes: 8.01
LLM model:  deepseek-v4-flash:cloud@v2

Ultragenyx gets FDA standard full approval for FAYUVI, first Sanfilippo type A treatment
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- FDA granted standard full approval of FAYUVI (UX111) for pediatric MPS IIIA (Sanfilippo syndrome type A) on Sept 17, 2026.
- FAYUVI is the first-ever FDA-approved treatment for Sanfilippo syndrome type A, a progressive fatal neurodegenerative disease.
- This is Ultragenyx's second gene therapy approval; company received a Priority Review Voucher upon approval.
- Approval covers pediatric patients with mucopolysaccharidosis type IIIA; trade name rebisufligene etisparvovec-hopf.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/0001515673-26-000006-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1515673/000151567326000006/rare-20260917.htm
  HTML page:      https://secwatch.observer/filing/0001515673-26-000006

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