Ultragenyx (NASDAQ:RARE) has submitted a marketing application to the FDA seeking approval of triheptanoin (UX007) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of inherited diseases in which the body is unable to metabolize long-chain fatty acids into energy. The indication has Rare Pediatric Disease Designation and Fast Track status.
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.
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