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Afinitor Approved for Rare Genetic Disorder

MONDAY, Nov. 1 -- Afinitor (everolimus) has received expanded approval from the U.S. Food and Drug Administration to treat a rare genetic disorder called subependymal giant cell astrocytoma (SEGA) that cannot be treated with surgery.

The disorder causes non-cancerous tumors on the brain and other parts of the body, including the eyes, lungs, liver, heart, skin and kidneys. It can be fatal if the brain tumors lead to complications, the FDA said in a news release. Symptoms may include learning disabilities, seizures, and lung and kidney disease.

Afinitor was first approved in March 2009 to treat kidney cancer among people in whom standard therapies had failed.

In clinical studies of people with SEGA, the most common side effects of Afinitor included infections of the upper respiratory tract, sinuses and ears, as well as mouth sores and fever.

Afinitor is marketed by Novartis, based in East Hanover, N.J.

More information

To learn more about this approval, visit the FDA.

Posted: November 2010

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