Ultragenyx Granted Orphan Drug Designation for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome

Industry News

According to the FDA, Ultragenyx Pharmaceutical has been granted Orphan Drug Designation for its triheptanoin for the treatment of fatty acid oxidation disorder (FAOD).  Ultragenyx is a Novato, California based biotechnology company focusing on development of therapeutics for rare genetic diseases.  UX007 (triheptanoin) is one of the drug candidates in the company’s pipeline being evaluated in Phase II study for treatment of long-chain fatty acid oxidation disorders (LC-FAOD).  FAOD is a group of rare hereditary metabolic disorders resulting from mutation in genes involved in fatty acid oxidation, a process that generate energy from fat. The inability to produce energy from fat can lead to depletion of glucose in the body, and severe liver, muscle, and heart disease, which can lead to early death.  Currently, there is no approved drug for this group of rare genetic diseases. Triheptanoin is a medium-chain triglyceride designed to provide substrate replacement for both fatty acid metabolism and the Krebs cycle and restore production of energy.  UX007 previously received FDA ODD for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS) in 2014.  Phase II clinical trials for both indications are ongoing.


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