The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Ultragenyx Netherlands for triheptanoin (Dojolvi), an oral liquid medicine used to treat adults and children with long-chain fatty acid oxidation disorders (LC-FAOD). The approval, granted on 30 October 2026, provides a new treatment option for patients living with these rare inherited metabolic disorders. However, the authorisation remains conditional, as the company must submit further evidence of the medicine’s efficacy and safety from an ongoing paediatric study to obtain full marketing authorisation.
Understanding Long-Chain Fatty Acid Oxidation Disorders
LC-FAOD refers to a group of rare inherited disorders that affect adults and children of all ages. People with these conditions cannot effectively use long-chain fatty acids as an energy source. Consequently, their bodies may struggle to produce sufficient energy, increasing the risk of serious clinical complications and frequent hospitalisations. These complications can include muscle breakdown, damage to the heart muscle and dangerously low blood sugar levels.
Triheptanoin Helps Prevent Cellular Energy Crises
Triheptanoin is an oral liquid medicine that patients take daily as part of a carefully controlled diet. It works by providing an alternative energy source to help prevent cellular energy crises that can lead to serious complications associated with LC-FAOD. The medicine is available only on prescription. Patients must follow their healthcare professional’s instructions regarding dosage and administration.
MHRA Highlights Importance of Access to Rare Disease Treatments
Julian Beach, MHRA Executive Director of Operations, said that improving patient access to high-quality, safe and effective medicines remains an important priority for the agency. He highlighted that patients with rare diseases often face significant barriers to accessing effective treatments. As reported by pharmabiz.com, the MHRA granted the authorisation after determining that the medicine met the required regulatory standards. Furthermore, the agency will continue to closely monitor the safety of triheptanoin following its approval.