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FDA approves drug for tuberous sclerosis complex kidney tumors

WASHINGTON (Reuters) – U.S. health regulators approved on Thursday Novartis AG’s Afinitor (everolimus) for the treatment of non-cancerous kidney tumors in patients with a rare genetic disease known as tuberous sclerosis complex.

The approval from the Food and Drug Administration makes Afinitor the first drug available specifically for non-cancerous tumors that do not require immediate surgery.

Tuberous sclerosis complex, or TSC, causes multiple tumors in both kidneys that compress normal tissue, leading to kidney failure and bleeding.

The disorder affects about 40,000 people in the United States, 70-80% of whom develop kidney problems. TSC also causes non-cancerous tumors in the brain and other organs.

Novartis’ treatment is a pill taken once daily that blocks the activity of mTOR kinase.

“This approval underscores the FDA’s commitment to the development of drugs for rare diseases with significant unmet medical needs,” according to a statement from Dr. Richard Pazdur, director of hematology and oncology products within FDA’s Center for Drug Evaluation and Research.

“It also represents another example of where an understanding of a disease’s underlying biology can lead to more selective drug development.”

Last September, Novartis released data from a late-stage study with 118 patients showing that 42% of TSC sufferers can experience responses including reduced tumor size and an absence of new tumors after taking the drug.