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# Ultragenyx Pharmaceutical Plans NDA Mid-2019
- URL: https://www.fdaweb.com/ultragenyx-pharmaceutical-plans-nda-mid-2019/
- Published: 2018-11-14T12:00:00.000Z
- Updated: 2026-09-15T00:44:32.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5142826

Ultragenyx Pharmaceutical has completed a pre-NDA meeting and plans to submit an NDA for UX007 for treating patients with long-chain fatty acid oxidation disorders in mid-2019\. The company says the meeting led to an agreement to include data from a Phase 2 company-sponsored study of UX007 in 29 patients, a long-term efficacy and safety extension study in 75 patients, a retrospective medical record review of 20 patients, data from 56 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of triheptanoin on cardiac function.

In the Phase 2 study, the data showed a 48.1% reduction in the mean annualized rate of major clinical events and a 50.6% reduction in the median annualized rate of major events after 78 weeks of treatment with UX007, the company says. The pre-NDA meeting also clarified the analyses and data presentations the agency will look for in the submission, it says.

Ultragenyx says UX007 is a highly purified, pharmaceutical-grade, synthetic, seven-carbon fatty acid triglyceride created via a multi-step chemical process. It is designed to provide patients with “medium-length, odd-chain fatty acids that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process,” it says. “Unlike typical even-chain fatty acids, UX007 can be converted to new glucose through the Krebs cycle, potentially providing an important added therapeutic effect, particularly when glucose levels are too low.”