FDA approves Afinitor for non-cancerous kidney tumors caused by rare genetic disease

            The U.S. Food and Drug Administration recently approved Afinitor (everolimus), the first drug approved specifically to treat non-cancerous kidney tumors (renal angiomyolipomas) not requiring immediate surgery in patients with tuberous sclerosis complex (TSC).

            TSC is a rare genetic disease that causes the growth of various non-cancerous tumors in the brain, kidney and other vital organs. It affects as many as 40,000 patients in the United States, with an estimated 70 to 80 percent developing kidney problems. TSC generally causes multiple tumors in both kidneys that compress normal kidney tissues as they increase in size, leading to kidney failure and bleeding.

            Afinitor blocks the uncontrolled activity of the mTOR kinase, which plays a critical role in the development and growth of the various non-cancerous tumors occurring in patients with TSC.

            The FDA granted Afinitor orphan drug designations to treat renal angiomyolipomas and a certain type of brain tumor called subependymal giant cell astrocytoma (SEGA) in patients with TSC in 2009. An orphan designation is given to a drug intended to treat a disease or condition affecting fewer than 200,000 patients in the United States and for which the drug, based on supporting data, has shown promise in the treatment of the disease.

            The drug’s application was granted priority review, a designation given to drugs that offer major advancements in treatment or provide a treatment where no adequate therapy exists. FDA completed review of this application in four months.

            The FDA has previously approved Afinitor 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 spread to other parts of the body.

Source: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm302048.htm