MHRA approves triheptanoin for rare inherited disorders that affect energy production
The oral liquid is for adults and children with long-chain fatty acid oxidation disorders, and the licence is conditional on further study results.
The UK's medicines regulator has approved triheptanoin, sold as Dojolvi, for adults and children with long-chain fatty acid oxidation disorders, a group of rare inherited conditions.
People with these disorders, known as LC-FAOD, cannot use long-chain fatty acids as an energy source, so they may have difficulty producing enough energy. That puts them at risk of major clinical events and frequent hospital stays. The Medicines and Healthcare products Regulatory Agency said the drug works by helping to prevent the cellular energy crises that can cause muscle breakdown, damage to the heart muscle and low blood sugar.
It is an oral liquid, taken every day as part of a carefully controlled diet, and is available only on prescription.
Why the approval is conditional
The MHRA has granted a conditional marketing authorisation to Ultragenyx Netherlands. This means a full authorisation depends on further evidence on efficacy and safety from a paediatric study that is still running. The agency says it will keep the medicine's safety under close review.
The most common side effects, which may affect more than 1 in 10 people, include stomach pain, diarrhoea and vomiting. Suspected side effects can be reported through the Yellow Card scheme.
The product information and patient leaflets are due on the MHRA Products website within seven days of the approval, which was announced on Friday.
