{
  "id": 2020809,
  "title": "Ultragenyx stock surges on FDA gene therapy approval",
  "url": "https://urgent.news/2026/08/19/ultragenyx-stock-surges-on-fda-gene-therapy-approval",
  "topic": "finance",
  "section": "Finance & Markets",
  "published": "2026-08-19T21:26:49.000Z",
  "source": {
    "name": "Investing.com",
    "slug": "investing-com",
    "url": "https://www.investing.com/news/stock-market-news/ultragenyx-stock-surges-on-fda-gene-therapy-approval-93CH-4868331"
  },
  "original_language": "en",
  "account": "Ultragenyx Pharmaceutical Inc.'s stock soared 14% in after-hours trading on Wednesday after the FDA granted accelerated approval to GENGLYCOS, the company's gene therapy for glycogen storage disease type Ia. This marks Ultragenyx's first gene therapy authorization and fifth overall FDA approval. GENGLYCOS, or DTX401, is specifically approved for adult and pediatric patients aged eight and older with GSDIa, a rare genetic metabolic disorder affecting 1,500-2,500 individuals in the U.S. The FDA's decision was based on positive results from the 48-week Phase 3 GlucoGene study involving 46 participants. The randomized, double-blind, placebo-controlled trial revealed a decrease in cornstarch requirements for patients treated with DTX401 compared to those receiving a placebo. GSDIa results from a deficiency of the enzyme required to convert glucose from the liver to the bloodstream, leading to patients needing continuous cornstarch intake to manage glucose levels and prevent life-threatening hypoglycemia. As part of the accelerated approval, Ultragenyx pledged to provide two years of safety and efficacy data from an open-label commercial treatment of 50 patients and 20 control patients through its GSDIa Disease Monitoring Program. The control group will comprise patients ineligible for GENGLYCOS due to anti-AAV8 antibodies. GENGLYCOS will be administered through a national network of Qualified Treatment Centers with specialized expertise in gene therapy. The therapy is produced at Ultragenyx's Gene Therapy Manufacturing Facility in Bedford, Massachusetts.",
  "summary": null,
  "key_points": [
    "Ultragenyx stock surges 14% after FDA accelerated approval",
    "GENGLYCOS gene therapy approved for GSDIa treatment",
    "Study showed DTX401 reduced cornstarch needs in patients"
  ],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 1,
    "also_reported_by": []
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}