FDA approves Novartis drug for tumors in patients with rare genetic disease

EAST HANOVER, N.J. — A drug made by Novartis has received approval from the Food and Drug Administration to treat benign kidney tumors related to tuberous sclerosis complex.



The FDA approved Afinitor (everolimus) tablets for treating the tumors, known as renal angiomyolipomas, in adults with TSC who don't require surgery. The drug already was approved to treat brain tumors known as subependymal giant cell astrocytoma, or SEGA, in patients with TSC.



The tumors occur in up to 80% of people with the disease, which affects between 25,000 and 40,000 people in the United States. TSC, a genetic disorder, can affect various parts of the body and causes disorders, such as skin lesions, seizures, brain swelling, kidney failure, developmental delays and behavioral problems. Onset typically occurs between the ages of 15 and 30 years, and the tumors can grow large enough to cause severe internal bleeding.



"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," Cincinnati Children's Hospital Medical Center nephrology chairman John Bissler said. "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."

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