The approval was given on the basis of results from prospective, open-label, single-arm Phase II study, in which 75% of patients (21 of 28) experienced a reduction of 30% or greater in the size of their largest SEGA and 32% (9 of 28) experienced a reduction of 50% or greater at six months relative to baseline.
In the study, 28 patients aged three years and above with evidence of SEGA growth initially received everolimus orally at a dose of 3mg/m daily or every other day.
Novartis Oncology president Herve Hoppenot said the approval of Votubia is significant for children and adults who have SEGA associated with tuberous sclerosis and, until now, have had limited treatment options.