Drug-resistant epilepsy drug Ontozry backed by NICE

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma's Ontozry.

The cost-effectiveness agency has backed Ontozry (cenobamate) as an option for treating focal onset seizures with or without secondary generalised seizures in adults with drug-resistant epilepsy that has not been adequately controlled with at least two prior antiseizure medicines.

It is recommended only if it is used as an add-on treatment, after at least one other add-on treatment has not controlled seizures, and treatment is started in a tertiary epilepsy service.

NICE notes in its final appraisal document (FAD) for the drug that many patients with this form of epilepsy may have to try a range of medicines, both alone and in combination, in order to control seizures. However third-line therapy is generally not very effective, and patients may have to resort to invasive therapy options like resective surgery and vagal nerve stimulation.

Epilepsy affects more than 600,000 people in the UK and for some, seizures can be life-threatening. It is estimated that around 1,000 people die in the UK every year because of the condition. Focal onset seizure is a type of epilepsy in which seizures start in one side of the brain, acconting for around 60% of all epilepsy cases.

"The FAD is a significant milestone for cenobamate and takes us closer towards reimbursement in England and Wales," commented Stuart Mulheron, general manager UK & Ireland, Angelini Pharma.

"We look forward to the full technology appraisal guidance (TAG) which is due to be published in early December 2021," he added.

An opinion from the Scottish Medicine Consortium (SMC) on the use of the drug by NHS Scotland is expected to be published early in 2022.

Italian group Angelini acquired Switzerland's Arvelle Pharma earlier this year in a deal valued at up to $960 million, a few days after Ontozry was recommended for approval by the EMA's human medicines committee.

The drug was eventually cleared in the EU in April, with the UK giving it a green light in June. The approvals were based on data from clinical trials CO17 and CO21 of Ontozry, involving 1,784 patients, which showed a 35% to 55% reduction in focal-onset seizures with treatment, depending on the dose used.

Cenobamate was originally developed by South Korean biotech SK Biopharmaceuticals, and was approved as Xcopri in the US in 2019. The drug made almost $17 million in US sales in the third quarter.