Posted by Michael Wonder on 30 Jun 2020
Ultragenyx announces U.S. FDA approval of Dojolvi (UX007/triheptanoin), the first FDA approved therapy for the treatment of long-chain fatty acid oxidation disorders
30 June 2020 - Fourth U.S. FDA approval for Ultragenyx in the past three years – all for rare diseases which previously had no approved therapies.
Ultragenyx today announced that the U.S. FDA has approved Dojolvi (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.
