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Novartis' Afinitor approved for noncancerous kidney tumors resulting from rare genetic disease

Thursday, April 26 2012 | Comments
Evidence Grade 0 What's This?
Novartis Pharmaceuticals Corp.'s Afinitor (everolimus) is the first drug to receive Food and Drug Administration approval specifically to treat noncancerous kidney tumors, or renal angiomyolipomas, that do not require immediate surgery in patients with tuberous sclerosis complex (TSC), a rare genetic disease that causes the growth of noncancerous tumors in the brain, kidney and other vital organs.

In 2009, the FDA granted Afinitor orphan drug designations in the treatment of renal angiomyolipomas and a certain type of brain tumor called subependymal giant cell astrocytoma (SEGA) in patients with TSC. In addition, the regulatory agency had granted the Afinitor marketing application for the renal angiomyolipoma indication a priority review.

This latest use of Afinitor was approved based on data from a double-blind trial of 118 patients who had tumors in both kidneys, many of whom had undergone a procedure to control bleeding from the tumors according to the FDA. Approximately two thirds of the patients were randomized to receive Afinitor and the remaining patients were treated with placebo.

The purpose of the trial was to measure the proportion of patients whose tumor shrank or disappeared after treatment. The trial was designed with a treatment period of as long as four years, and patients in the placebo group were offered treatment with Afinitor if their tumors grew during the study.

With half of the patients receiving treatment for less than eight months, 42 percent of those who received Afinitor showed a substantial reduction in tumor size that lasted, on average, more than five months. None of the placebo-treated patients experienced tumor shrinkage, the FDA noted.   

Afinitor is already approved to treat patients with advanced renal cell carcinoma that has progressed after treatment with other cancer therapies; SEGA associated with TSC in patients who require treatment but are not candidates for surgical removal of the tumor; and progressive neuroendocrine tumors located in the pancreas that cannot be removed by surgery or that have metastasized.

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