The U.S. Food and Drug Administration has announced that is has approved Afinitor (everolimus) as 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 organs. As many as 40,000 people in the US are affected annually, and as many as 4 in 5 of them develop kidney problems. TSC is a disease that can cause the development of several tumors that can ultimately lead to kidney failure.
Afinitor is a daily oral medication that blocks the uncontrolled activity of the mTOR kinase protein, which plays a critical role in the development and growth of the various non-cancerous tumors occurring in patients with TSC.
The FDA granted orphan drug status to Affinitor to treat renal angiomyolipomas and a certain type of brain tumor called subependymal giant cell astrocytoma (SEGA) in patients with TSC in 2009. Later the application to the FDA for approval was granted priority review and the FDA finished the review in four months' time.
“This approval underscores the FDA’s commitment to the development of drugs for rare diseases with significant unmet medical needs,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the 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.”
Source: FDA
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