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# NDA for Ultragenyx Fatty Acid Oxidation Drug
- URL: https://www.fdaweb.com/nda-for-ultragenyx-fatty-acid-oxidation-drug/
- Published: 2019-10-14T12:00:00.000Z
- Updated: 2026-09-15T01:56:15.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145237

FDA has accepted for review an Ultragenyx Pharmaceutical NDA for UX007 (triheptanoin) for treating long-chain fatty acid oxidation disorders, a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The company says UX007 is a “highly purified, pharmaceutical-grade, medium-chain triglyceride consisting of three seven-carbon fatty acids on a glycerol backbone created via a multi-step chemical process.” The agency has set a standard review target date of 7/31/2020\. The company says FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application.

The NDA is based on data from a company-sponsored Phase 2 study of UX007 in 29 patients, a long-term safety and efficacy extension study in 75 patients including 20 patients who were previously naive to UX007, a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect on cardiac function, according to the company.