Ultragenyx Pharmaceutical Inc. said the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization to Dojolvi (triheptanoin), its treatment for long-chain fatty acid oxidation disorders (LC-FAOD). This is the drug’s first approval in Europe, several years after it was cleared in the United States. The oral liquid is for adults and children with these rare inherited metabolic conditions.
The authorization requires Ultragenyx to submit additional efficacy and safety data from an ongoing pediatric study. UK patients’ access through the National Health Service will also depend on an appraisal by the National Institute for Health and Care Excellence (NICE), which is not expected to begin until October 2027.
Dojolvi generated $45 million in sales in the first half of the year.