MHRA Grants Conditional Approval to Dojolvi for Adults and Children with LC-FAOD – Ultragenyx
On 30 October 2026, the MHRA granted Dojolvi (triheptanoin), an oral liquid, conditional marketing authorisation to treat adult and paediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). The authorisation was granted to Ultragenyx Netherlands.
Further evidence of its efficacy and safety from an ongoing paediatric study is required for full marketing authorisation.
LC-FAOD is a group of rare inherited disorders affecting adults and children of all ages. Patients may have difficulty producing 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 hospitalisations.
Triheptanoin is a liquid that is taken daily by mouth, as part of a carefully controlled diet. It can only be obtained with a prescription, and patients should always take this medicine exactly as directed by their healthcare professional.
The medicine works by helping to prevent energy crises in the cells that can cause muscle breakdown, damage to the heart muscle and low blood sugar.
The most common side effects of the medicine (which may affect more than 1 in 10 people) include stomach pain, diarrhoea, and vomiting.
Source: MHRA
