{"schema_version":"secwatch.filing_event.v1","accession":"0001193125-26-034474","form_type":"8-K","ticker":"RARE","cik":"0001515673","company_name":"Ultragenyx Pharmaceutical Inc.","filed_at":"2026-02-03T23:59:59+00:00","discovered_at":"2026-05-14T18:02:35.289208+00:00","generated_at":"2026-05-16T05:18:45.691611+00:00","sec_items":["8.01"],"event_type":"other_material","sentiment":"positive","materiality_score":0.85,"calibrated_materiality_score":0.85,"confidence":"high","headline":"Ultragenyx UX111 gene therapy shows significant cognitive benefit in MPS IIIA; BLA resubmitted with PDUFA Q3 2026","bullets":["Children under 2 treated with UX111 showed +23.2 point improvement in Bayley-III cognitive raw score vs natural history (p<0.0001).","81.5% of overall efficacy set achieved ≥50% reduction in CSF heparan sulfate; median reduction 63.98% (p<0.001).","Functional retention observed: all older children retained communication; 9/10 retained ambulation and self-feeding.","UX111 well-tolerated with median follow-up 4.8 years; no TMA, myocarditis, DRG toxicity, or malignancy.","BLA resubmitted to FDA seeking accelerated approval; PDUFA date expected in Q3 2026."],"urls":{"canonical":"https://secwatch.observer/filing/0001193125-26-034474","json":"https://secwatch.observer/filing/0001193125-26-034474.json","markdown":"https://secwatch.observer/filing/0001193125-26-034474.md","text":"https://secwatch.observer/filing/0001193125-26-034474.txt","edgar_index":"https://www.sec.gov/Archives/edgar/data/1515673/000119312526034474/0001193125-26-034474-index.htm","edgar_primary_document":"https://www.sec.gov/Archives/edgar/data/1515673/000119312526034474/rare-20260203.htm"},"model":{"generated_by":"deepseek-v4-flash:cloud@v2","generated_at":"2026-05-16T05:18:45.691611+00:00"},"review":{"review_status":"machine_generated","human_reviewed":false,"corrected":false,"correction_note":null,"correction_timestamp":null,"superseded_by":null,"related_filings":[]},"source_grounded_claims":[],"license":"Source filings: public domain (SEC EDGAR). Summaries (headline + bullets): CC-BY-4.0; attribute https://secwatch.observer"}