A Novartis Phase III clinical trial has shown that Everolimus significantly controls seizures in TSC patients when taken in addition to the patients' usual anti-epileptic drugs (AEDs). The results of the EXIST-3 (epilepsy) study were presented to a meeting of the American Academy of Neurology (ANN) on 20th April 2016.
Around 85% of individuals are affected by epilepsy at some point in their lives. Around two thirds of these patients have seizures that resistant to treatment by available therapies.
In the EXIST-3 study, 366 patients with TSC and treatment-resistant seizures were randomised to received low dose Everolimus, high dose Everolimus or a placebo. The reduction in seizure frequency was found to be significantly greater in patients receiving the Everolimus - 29.3% fewer in the low dose group and 39.6% fewer seizures in the high dose group.
Before Everolimus can be considered for NHS funding the results of the study will need to be reviewed by the medicines regulators, the European Medicines Agency (the agency that issues licences for medicines that have been proven effective and safe).