Patients with kidney tumours linked to the rare condition Tuberous Sclerosis Complex (TSC) will now, for the first time, have access to an effective non-surgical treatment option following the launch of Novartis' Votubia in the UK.
TSC is a genetic disorder that is thought to affect around 6,600 people in the UK. Eighty percent of patients develop kidney tumours, which potentially cause debilitating symptoms and can significantly harm kidney function.
Novartis won European approval for its once-daily pill in October last year for TSC patients with renal angiomyolipoma at risk of complications (based on factors such as tumour size or presence of aneurysm) but not requiring immediate surgery.
The decision came on the back of data from the Phase III EXIST-2 trial, which found that 42% of patients taking Votubia (everolimus) experienced a response - reducing the size of the tumour - versus 0% of those taking a placebo.
Until now, treatment options were limited to surgical interventions, usually 'embolisation' or the full or partial removal of the affected kidney, and so the availability of Votubia "has the potential to redefine the way people with TSC kidney tumours are treated”, noted Chris Kingswood, Consultant Renal Physician at the Royal Sussex County Hospital in Brighton.
But the drug doesn't come cheap; a PR representative for Novartis told PharmaTimes UK News Online that the average dose of Votubia is usually 10mg a day, which equates to a cost of £2,970 a month.