UK regulator conditionally authorises triheptanoin for rare inherited metabolic disorders
MHRA announced a conditional authorisation for Dojolvi for adults and children with long-chain fatty acid oxidation disorders; further study evidence is required.
What the record says
The UK Medicines and Healthcare products Regulatory Agency announced on October 30 that it has conditionally authorised an oral liquid form of triheptanoin, branded Dojolvi, for adult and paediatric patients with long-chain fatty acid oxidation disorders.
MHRA says a full marketing authorisation requires further evidence of efficacy and safety from an ongoing paediatric study. The medicine is prescription-only and is used as part of a carefully controlled diet under healthcare-professional direction. The regulator says it will keep safety under review.
Source details and supporting locations.
Practical next step
Read MHRA’s announcement and discuss individual treatment questions with a qualified healthcare professional.
Limits and what is not established
This records a conditional regulatory decision, not a cure, full authorisation, availability guarantee or individual treatment recommendation.
Sources checked October 30, 2026. Later notices may change instructions or arrangements.
Original sources
Medicines and Healthcare products Regulatory Agency, GOV.UK: original information