Triheptanoin (Dojolvi) approved to treat adults and children with a group of rare inherited disorders
MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders.
The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 30 October 2026, approved an oral liquid form of triheptanoin (Dojolvi) to treat adult and paediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). The product has a conditional marketing authorisation because further evidence of its efficacy and safety from an ongoing paediatric study is required for a full marketing authorisation.
LC-FAOD is a group of rare inherited disorders in adults and children of all ages.
Patients with LC-FAOD may have difficulty producing enough energy because of their body’s inability to use long-chain fatty acids as an energy source. This puts them at risk of major clinical events resulting in frequent hospitalisations.
Julian Beach, MHRA Executive Director, Operations, said:
“Enabling patient access to high quality, safe and effective medical products is important to us.
“Patients living with rare diseases often face significant barriers in accessing effective treatments and we’re assured that the appropriate regulatory standards for the approval of this medicine have been met.
“As with all products, we will keep the safety of triheptanoin under close review.”
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.
Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the Yellow Card scheme.
Notes to editors
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The new marketing authorisation was granted on 30 October 2026 to Ultragenyx Netherlands.
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More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
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The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe. All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
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The MHRA is an executive agency of the Department of Health and Social Care.
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For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.