Novartis Pharmaceuticals Corporation announced today that the FDA approved Afinitor (everolimus) tablets 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 (EXamining everolimus In a Study of TSC) 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).

“Renal angiomyolipomas are one of the greatest causes of morbidity and mortality in adult TSC patients and can be one of the most challenging aspects of the disease to treat,” said John Bissler, MD, Clark D. West Endowed Chair of Nephrology at Cincinnati Children’s Hospital Medical Center. “Today marks an important step for the TSC community, as Afinitor is now the only approved medicine to reduce the kidney tumor burden in these patients.” 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(1). The tumors can be difficult to manage as they often form in both kidneys(5,6). In addition, skin lesions occur in more than 90% of patients with TSC(7). They may develop in infancy, can become more prevalent with age and cause disfigurement(1,5).

“With this FDA approval, Afinitor becomes the first medical option to treat two of the most debilitating manifestations of this challenging, lifelong disease – kidney tumors called renal angiomyolipomas and brain tumors known as SEGAs,” said Herve Hoppenot, President, Novartis Oncology. “This approval further strengthens our commitment to address unmet needs in TSC as we continue to research everolimus and mTOR inhibition across other manifestations of the disease.”