Decision marks the third TSC-related indication for Votubia in the EU.

Novartis has announced that the European Commission has approved Votubia (everolimus) dispersible tablets as an adjunctive treatment for patients aged 2 years and older whose refractory partial-onset seizures, with or without secondary generalization, are associated with tuberous sclerosis complex (TSC). Votubia is now the first approved pharmacologic therapy in all 28 member states of the European Union (EU), plus Iceland and Norway, specifically for the treatment of refractory partial-onset seizures associated with TSC.

"With this latest approval of Votubia in the EU, patients with TSC suffering from refractory partial-onset seizures - one of the most debilitating manifestations of TSC - now have a new therapeutic option to address a critical unmet need," said Bruno Strigini, CEO, Novartis Oncology. "This is a welcome advance and an important milestone in our ongoing commitment to improving care for this patient community."

The EU approval of Votubia was based on efficacy and safety data from a pivotal Phase III study (EXIST-3), 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 18 weeks of study core phase. The youngest patient enrolled was 2 years of age. Seizure response rate was significantly greater with Votubia low exposure (LE) and high exposure (HE) 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 >=15% in Votubia LE/HE arms included stomatitis, diarrhea, nasopharyngitis, upper respiratory tract infection, and pyrexia.