The FDA has approved everolimus (Afinitor) for the treatment of renal angiomyolipomas and tuberous sclerosis complex (TSC) in adult kidney tumor patients who do not require immediate surgery.

In a double-blind, randomized, placebo-controlled Phase 3 trial, 42% of patients on everolimus experienced an angiomyolipoma response compared with 0% on placebo. It also demonstrated a 26% response rate to skin lesions, a key concern for the TSC patients compared with to 0% with a placebo.

Everolimus is manufactured by Novartis of East Hanover, N.J. While it previously received FDA approval for the treatment of subependymal giant cell astrocytoma associated with TSC, it will be the first option for the treatment of both kidney and brain tumors.

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“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.