UX007 (a highly purified, synthetic seven carbon fatty acid triglyceride) is an investigational pharmaceutical-grade, specially designed synthetic triglyceride compound, created via a multi-step chemical process and purified, which is in development for long-chain fatty acid oxidation disorders (LC-FAOD).
LC-FAODs are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body, and serious liver, muscle, and heart disease, which can lead to hospitalizations or early death. LC-FAODs are included in newborn screening panels across the U.S. and in certain European countries.
LC-FAOD patients are currently treated with the avoidance of fasting, low-fat/high carbohydrate diets, carnitine, and medium-chain triglyceride (MCT) oil, a medical food product. Despite current therapy, many patients have significant metabolic events including hospitalizations and mortality due to LC-FAOD.
Ultragenyx has licensed rights to UX007 from Baylor Research Institute.
Mechanism of Action
UX007 is intended to provide patients with medium-length, odd-chain fatty acids that can bypass the genetic block in long-chain fatty acid metabolism. Due to its odd-chain properties, UX007 is metabolized into ketones that replace deficient intermediates in the Krebs cycle, a key energy-generating process. UX007 can also support production of glucose (gluconeogenesis) and is glycogen sparing. Together, the substrates produced by UX007 during metabolism are intended to improve energy production in LC-FAOD patients.
News & Other Information
- Ultragenyx Granted Additional Orphan Drug Designations for Triheptanoin (link)
- Case series of five infant FAOD patients with cardiomyopathy treated with triheptanoin under expanded access - SSIEM 2015 (pdf)
- Retrospective chart review of triheptanoin in FAOD patients - ICIEM 2013 (pdf)