MHRA approves oral liquid medicine triheptanoin for rare inherited energy disorders
The Medicines and Healthcare products Regulatory Agency (MHRA) approved an oral liquid form of triheptanoin, sold under the brand name Dojolvi, on 30 October 2026. The prescription medicine targets adult and paediatric patients living with long-chain fatty acid oxidation disorders (LC-FAOD). These rare inherited conditions prevent the body from using long-chain fatty acids to produce enough energy, putting patients at risk of severe medical events and frequent hospitalisation. Taken daily by mouth as part of a strictly controlled diet, the treatment works by helping prevent cellular energy crises that cause muscle breakdown, heart muscle damage, and low blood sugar. The MHRA granted a conditional marketing authorisation to Ultragenyx Netherlands, meaning full approval requires additional evidence of safety and efficacy from an ongoing paediatric study. Common side effects affect more than 1 in 10 people and include stomach pain, diarrhoea, and vomiting.
What was announced
On 30 October 2026, the Medicines and Healthcare products Regulatory Agency approved an oral liquid medicine called triheptanoin, sold under the brand name Dojolvi. The marketing authorisation was granted to Ultragenyx Netherlands.
The MHRA issued a conditional marketing authorisation for treating both adult and child patients. A full approval will require the company to supply further evidence of efficacy and safety from an active study in children.
What it means
Long-chain fatty acid oxidation disorders are rare genetic conditions affecting adults and children. People with these disorders cannot process long-chain fatty acids for energy, leaving them vulnerable to energy crises that can cause heart muscle damage, muscle breakdown, and low blood sugar. This frequently leads to hospital stays.
Triheptanoin works by helping prevent these energy breakdowns inside cells. It must be taken daily by mouth, strictly under prescription and alongside a carefully controlled diet.
Like all medicines, triheptanoin can cause side effects. The most common side effects, which may affect more than 1 in 10 patients, include stomach pain, diarrhoea, and vomiting. The MHRA asks patients to report any suspected side effects to the Yellow Card scheme.
What the announcement does not tell us
The announcement does not state when triheptanoin will be available to patients or what its list price will be. It does not clarify whether the treatment will be funded by the NHS in the UK.
The release does not specify how many patients in the UK have these disorders or how long the ongoing paediatric trial will take to complete. It also does not explain the exact timeline for full marketing authorisation.
How this was written
This analysis was written with AI from the official announcement below, using only facts that announcement states. The quoted wording is gov.uk: Medicines and Healthcare products Regulatory Agency’s own. Always check the original before acting on anything here.
MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders.
Opens www.gov.uk
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