EU approves Votubia (everolimus) as adjunctive treatment for partial-onset seizures associated with tuberous sclerosis complex- Novartis

Novartis announced that the European Commission has approved Votubia (everolimus) dispersible tablets as an adjunctive treatment for patients aged two years and older whose refractory partial-onset seizures, with or without secondary generalization, are associated with tuberous sclerosis complex (TSC). The EU approval of Votubia was based on efficacy and safety data from a pivotal Phase III study (EXIST-3: EXamining everolimus In a Study of TSC), which found that when used as an adjunctive therapy, Votubia significantly reduced the frequency of refractory partial-onset seizures associated with TSC compared to placebo.

Efficacy and safety of two trough exposure concentrations of Votubia, 3-7 ng/mL (low exposure) and 9-15 ng/mL (high exposure) were assessed. Patients in all treatment arms concomitantly received one to three anti-epileptic drugs (AEDs) during the eighteen weeks of study core phase. The youngest patient enrolled was two years of age. Seizure response rate (at least 50% reduction) was significantly greater with Votubia low exposure (LE) and high exposure vs placebo. The median percentage reduction from baseline in seizure frequency was also significantly greater among patients randomized to Votubia LE and HE vs placebo. The most common all-grade adverse events (AEs) of any cause reported during the core phase at frequencies of at least 15% in Votubia LE/HE arms included stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia