Triheptanoin (Dojolvi) approved to treat adults and children with a group of rare inherited disorders
What was announced
MHRA approves triheptanoin to treat adult and paediatric patients with a group of rare inherited disorders.
Key points, in the publisher's words
- 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.
Who and where
UK bodies of power
- Medicines and Healthcare products Regulatory Agency (executive agency) · more news
- Department of Health and Social Care (UK government department) · more news
Where this sits in the 10 levels of power
Topics, as GOV.UK files them: Marketing authorisations, variations and licensing guidance.
Receipt
- Original: https://www.gov.uk/government/news/triheptanoin-dojolvi-approved-to-treat-adults-and-children-with-a-group-of-rare-inherited-disorders
- Published 2026-10-30 14:55:53 UTC
- Read by this site 2026-10-30 15:00:13 UTC from GOV.UK
- Saved copy: receipt 5006, with its SHA-256 fingerprint
Contains public sector information licensed under the Open Government Licence v3.0.
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