The Medicines and Healthcare products Regulatory Agency (MHRA) approved an oral liquid form of triheptanoin (Dojolvi) on 30 October 2026 for adult and paediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare inherited conditions affecting people of all ages. The authorisation is conditional, with further evidence of efficacy and safety from an ongoing paediatric study required before a full marketing authorisation.

According to the MHRA, people with LC-FAOD may struggle to produce enough energy because their bodies cannot use long-chain fatty acids as an energy source, putting them at risk of major clinical events and frequent hospitalisation. The medicine is taken daily by mouth as part of a carefully controlled diet and is available only on prescription. It works by helping prevent energy crises in cells that can cause muscle breakdown, heart muscle damage and low blood sugar. Common side effects, which may affect more than 1 in 10 people, include stomach pain, diarrhoea and vomiting.

Julian Beach, the MHRA's Executive Director, Operations, said the agency was assured that appropriate regulatory standards had been met and that triheptanoin's safety would remain under close review. The authorisation was granted to Ultragenyx Netherlands, and product information is expected on the MHRA Products website within 7 days.