RegulatoryOfficial

MHRA grants conditional approval to triheptanoin (Dojolvi) for long-chain fatty acid oxidation disorders

🇬🇧 MHRA · Fri 30 Oct 2026

The UK regulator has authorised an oral liquid triheptanoin for adults and children with LC-FAOD, pending further efficacy and safety evidence from an ongoing paediatric study.

The Medicines and Healthcare products Regulatory Agency (MHRA) approved an oral liquid formulation of triheptanoin (Dojolvi) on 30 October 2026. It is indicated for adult and paediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). The marketing authorisation was granted to Ultragenyx Netherlands.

The authorisation is conditional. The MHRA said it needs further evidence on efficacy and safety from an ongoing paediatric study before a full marketing authorisation can be considered.

LC-FAOD is a group of rare inherited disorders affecting people of all ages. Patients have difficulty using long-chain fatty acids as an energy source, which puts them at risk of major clinical events and frequent hospitalisation. According to the agency, triheptanoin helps prevent cellular energy crises that can cause muscle breakdown, damage to the heart muscle and low blood sugar.

Triheptanoin is taken daily by mouth as part of a carefully controlled diet and is available on prescription only. The most common side effects, which may affect more than 1 in 10 people, are stomach pain, diarrhoea and vomiting. Suspected adverse reactions should be reported to the Yellow Card scheme.

Julian Beach, MHRA Executive Director, Operations, said: “As with all products, we will keep the safety of triheptanoin under close review.” The Summary of Product Characteristics and patient information leaflet are due on the MHRA Products website within seven days of approval.

What it means for your team

  • Pharmacovigilance teams should note the conditional status and the MHRA's stated intention to keep safety under close review; Yellow Card reporting is encouraged for suspected side effects.
  • Regulatory affairs teams should track the ongoing paediatric study, as its results underpin any move to a full marketing authorisation.
  • Quality and labelling teams should check the SmPC and patient leaflet once published on the MHRA Products website, expected within seven days of approval.
  • Market-access teams should note that the product is prescription-only and used alongside a carefully controlled diet; the announcement gives no pricing or reimbursement information.

Official source: MHRA ↗

Article written by VigiEye from the official source (MHRA). AI-assisted; check the source before acting.

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