Novartis has received the green light from the European Commission for Volubia for the treatment of patients with renal angiomyolipoma associated with tuberous sclerosis complex.
Specifically, the approval for Volubia (everolimus) is for aforementioned patients who are at risk of complications (based on factors such as tumour size or presence of aneurysm) but who do not require immediate surgery. Novartis noted that this marks the first approval of a medical treatment in this patient population in Europe.
The thumbs-up is based on data from the Phase III EXIST-2 trial, which found that 42% of patients taking everolimus experienced an angiomyolipoma response versus 0% on placebo. Median time to angiomyolipoma progression was 11.4 months in the placebo arm and was not reached in the Volubia arm.
Novartis cited Chris Kingswood of the Royal Sussex County Hospital, UK, who said renal angiomyolipomas affect the vast majority of patients with TSC "and over time can cause debilitating symptoms, a decline in renal function, and possibly even the need for kidney transplant or dialysis". He added that this approval "represents a major advance in the treatment of the disease in Europe". The drug is already approved for this indication in the USA as Afinitor.
Last year, Volubia was approved in Europe for patients aged three and older with subependymal giant cell astrocytoma (SEGA), a benign tumour type.