Everolimus (trade name Votubia®) has been approved by the regulators for use in the European Union as a treatment for refractory partial-onset seizures in children (aged 2 years and older) and adults with TSC.The term “refractory” refers to seizures that have stopped responding to other available anti-epileptic medications.Licenses are granted if the drug has been shown to work in clinical trials and if its side-effects are considered to be acceptable, and outweighed by the benefit of the drug. In granting a licence for Everolimus to treat AMLs the EMA considered evidence from the EXIST-3 clinical trial.
This study found that everolimus significantly reduced the frequency of refractory partial-onset seizures associated with TSC compared to a placebo (dummy
treatment).In those receiving everolimus, the frequency of seizures reduced by around a third. The most common side effects experienced by study
participants included mouth ulcers, diarrhoea, upper respiratory tract infection, and raised temperatures.
Everolimus for the treatment of refractory epilepsy is not yet routinely available on the NHS in the UK.
This means that decisions on whether Everolimus can be prescribed for epilepsy on the NHS will be made at the local level for the time being. Requests
for funding for Everolimus would need to be made by your specialist.If you would like to find out whether Everolimus might be a suitable treatment
for yourself or a person with TSC for whom you care, you should ask to be referred by your GP to a TSC clinic.