FDA approves Afinitor for treatment of Renal Angiomyolipomas

The FDA has on 26 April 2012 approved Afinitor (everolimus) tablets from Novartis for the treatment of adult patients with kidney tumors known as Renal Angiomyolipomas and Tuberous Sclerosis Complex (TSC), who do not require immediate surgery. This marks the first approval of a medical treatment in this patient population. The accelerated approval was based on the Phase III EXIST-2 trial, which found that 42% of patients on everolimus experienced an angiomyolipoma response versus 0% of patients in the placebo arm (p<0.0001. the time to angiomyolipoma progression was also statistically significantly longer in patients on everolimus p><0.0001). among the 97 of trial patients with skin lesions one of the key concerns for the majority of patients with tsc a 26 response rate was seen with everolimus versus 0 with placebo p="0.0011)." up to 80 of patients with tsc a genetic disorder that may cause non-cancerous tumors to form in vital organs will develop renal angiomyolipomas. typical onset occurs between the ages of 15 and 30 and prevalence increases with age. over time these tumors may grow large enough to cause severe internal bleeding require emergency surgical interventions such as embolization and nephrectomy or lead to kidney failure. the tumors can be difficult to manage as they often form in both kidneys. in addition skin lesions occur in more than 90 of patients with tsc. they may develop in infancy can become more prevalent with age and cause disfigurement.>