Good news · Health & science · 3 November 2026
MHRA approves new liquid treatment for rare metabolic disorder
The MHRA has approved an oral liquid form of triheptanoin, known as Dojolvi, for adults and children with long-chain fatty acid oxidation disorders. The medicine is taken daily as part of a controlled diet. Full authorisation is pending further evidence from an ongoing paediatric study.
The Medicines and Healthcare products Regulatory Agency has approved Dojolvi, an oral liquid medicine containing triheptanoin. The agency confirmed that the product is intended for use in both adult and paediatric patients of all ages who live with long-chain fatty acid oxidation disorders. This approval allows the medicine to be prescribed to help manage the condition.
Julian Beach, MHRA Executive Director, Operations, stated: "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." The marketing authorisation was granted to Ultragenyx Netherlands on 30 October 2026. Detailed product information, including the Summary of Product Characteristics and Patient Information leaflets, will be published on the MHRA website within 7 days of the approval.
The approval is conditional because further evidence of efficacy and safety is required from an ongoing paediatric study for full marketing authorisation. The medicine works by helping to prevent energy crises in cells, which can otherwise lead to muscle breakdown, heart damage, and low blood sugar. It is taken daily by mouth as part of a controlled diet and requires a prescription.
Common side effects associated with the treatment include stomach pain, diarrhoea, and vomiting, affecting up to 1 in 10 people. The MHRA has indicated that the safety of triheptanoin will remain under close review. The announcement does not specify the cost of the medicine or the timeline for the completion of the ongoing study.
Written by StreetOwl from MHRA’s own announcement. It was drafted by StreetOwl’s own model, every figure and quotation was checked by code against the announcement, and it was read and approved by Michael Rossi before it went up. It says what was announced, not what anyone should do about it.
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