Ultragenyx gets first Euro nod for rare disease drug Dojolvi

Several years after it was approved in the US, Ultragenyx's therapy for long-chain fatty acid oxidation disorders (LC-FAOD), Dojolvi, has been greenlit in the UK.

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted a conditional marketing authorisation to Dojolvi, an oral liquid formulation of triheptanoin, to treat adult and paediatric patients with LC-FAOD. The UK approval is believed to be the first for the drug in Europe.

LC-FAOD is a group of rare, inherited genetic metabolic conditions where the body cannot convert long-chain fatty acids into energy. That means that during periods of fasting, when the body switches to running on fat reserves, rather than carbohydrates, organs that need a lot of energy, like the brain, heart, and muscles, stop working properly. Moreover, unprocessed fats and their byproducts can accumulate in tissues and organs, causing toxicity.

Management of the disorders currently relies on careful management of dietary fat intake, with frequent meals, avoiding prolonged fasting, low-fat, or controlled diets, and supplementation with medium-chain triglyceride (MCT) oil, which bypasses the defective LC-FA breakdown pathway and provides a safe, usable source of energy for the body.

Dojolvi is a pharmaceutical-grade form of MCT, with important differences in composition that improve its efficacy, and is taken daily as part of a carefully controlled diet. MCT oils can become less effective over time and sometimes fail to prevent severe metabolic crises like cardiomyopathy.

"Enabling patient access to high-quality, safe, and effective medical products is important to us," said MHRA's executive director of operations, Julian Beach.

"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," he added. "As with all products, we will keep the safety of triheptanoin under close review."

Patients with LC-FAOD hoping for access to Dojolvi will still have to wait for the conclusion of an appraisal by reimbursement authority NICE, unless they are able to pay for a private prescription. An update on the NICE process, posted to the agency's website in August, said the appraisal is "anticipated to begin in October 2027."

Dojolvi was approved in the US in 2020 and was also launched in Japan earlier this year. Ultragenyx recorded sales of $45 million in the first half of this year, and has said it expects full-year revenues from the drug to reach $100 million to $110 million.