The European Commission (EC) has approved Votubia® (everolimus) for patients, aged three years and above, with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC), who require therapeutic intervention but are not amenable to surgery.
Prior to the approval of Votubia, an oral medication, brain surgery was the only treatment option for children and adults in the European Union with SEGA associated with TSC.
The approval from the EC follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in June and is based on a prospective, open-label, single-arm, Phase II study of 28 patients.
Results from this trial showed that 78% of patients (21 of 27) experienced a reduction of 30% or greater in the size of their largest SEGA and 33% (9 of 27) experienced a reduction of 50% or greater at six months relative to baseline.
An additional placebo-controlled Phase III study examining this patient population was recently presented at the International TSC Research Conference in July. This trial met its primary endpoint of SEGA response rate.
“This approval is an important step forward in managing SEGAs associated with tuberous sclerosis complex, as surgery was previously the only available treatment option in the EU for these patients,” said Dr Sergiusz Jozwiak, Professor of the Department of Child Neurology at the Children’s Memorial Health Institute in Warsaw, Poland.
“As the first approved medication for this patient community, Votubia will help fill a critical unmet treatment need.”