Britain's Medicines and Healthcare products Regulatory Agency (MHRA) authorized triheptanoin for adults and children with long-chain fatty acid oxidation disorders, allowing treatment while more pediatric safety and efficacy evidence is gathered.
The agency granted Ultragenyx Netherlands a conditional marketing authorization on Oct. 30. Results from an ongoing study in children must provide further safety and efficacy evidence before the medicine can receive full authorization, the MHRA said.
Long-chain fatty acid oxidation disorders are rare inherited conditions that disrupt the body's use of long-chain fatty acids as fuel. The MHRA said patients may struggle to produce enough energy and face serious medical events and repeated hospital stays.
Sold as Dojolvi, triheptanoin is an oral liquid taken each day with a carefully managed diet and is available only by prescription. The MHRA says it may help avert energy crises in cells that can lead to muscle breakdown, heart muscle damage and low blood sugar.
Stomach pain, diarrhea and vomiting are among side effects that may affect more than 10% of patients, the agency said. Julian Beach, MHRA Executive Director, Operations, said the regulator would continue reviewing the medicine's safety. The agency plans to publish prescribing information and patient leaflets on its MHRA Products website within seven days of approval.
