UK Conditionally Clears Ultragenyx's Triheptanoin to Treat Rare Inherited Disorders

MT Newswires · 3d ago
11:31 AM EDT, 10/30/2026 (MT Newswires) -- The UK conditionally approved Ultragenyx Pharmaceutical (UP0.F) unit Ultragenyx Netherlands' oral liquid form of triheptanoin for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders, or LC-FAOD. The British Medicines and Healthcare products Regulatory Agency said Friday that the full marketing authorization for triheptanoin, or Dojolvi, is conditional on further efficacy and safety results from an ongoing pediatric study. LC-FAOD is a group of rare inherited disorders that can put patients at risk of major clinical events due to the body's inability to use long-chain fatty acids as an energy source.