Votubia/Afinitor success in treatment of Renal Angiomyolipoma reported in The Lancet

Data published in The Lancet showed that patients on Afinitor (everolimus) tablets from Novartis, with non-cancerous kidney tumors known as Renal Angiomyolipomas associated with Tuberous Sclerosis Complex (TSC) experienced a significant reduction in tumor size and the absence of tumor progression. Additionally, a recent issue of The Lancet featured results from a separate everolimus trial demonstrating a reduction in the size of non-cancerous brain tumors known as subependymal giant cell astrocytomas (SEGAs) associated with TSC.

TSC is a genetic disorder that may cause non-cancerous tumors to form in vital organs, including the kidney and brain. Known as Votubia in the European Union and Switzerland for the treatment of certain patients with TSC, everolimus is the first and only medication for adult patients with these kidney tumors associated with TSC who do not require immediate surgery, and separately for pediatric and adult patients with SEGAs who require therapeutic intervention but are not amenable to surgery.

The Phase III EXIST-2 trial published in The Lancet reported that 42% of patients taking everolimus experienced an angiomyolipoma response versus 0% of patients in the placebo arm (p<0.0001). see bissler j et al. everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis: a multicentre randomised double-blind placebo-controlled trial. the lancet 2012: 380.>