FDA accepts Ultragenyx’s triheptanoin NDA for rare oxidation disorders

The FDA accepts for review Ultragenyx’s (NASDAQ:RARE) marketing application seeking approval of UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of inherited diseases in which the body is unable to convert long-chain fatty acids into energy.

The agency’s action date is July 31, 2020. An advisory committee meeting is not planned.

UX007 is a purified pharmaceutical-grade form of triheptanoin, a triglyceride compound, that provides patients with medium-length odd-chain fatty acids which increase the intermediate substrates in the Krebs cycle, a key energy-generating process, which produces new glucose.

https://seekingalpha.com/news/3505469-fda-accepts-ultragenyxs-triheptanoin-nda-rare-oxidation-disorders