FDA approves new indication for Afinitor

The U.S. Food and Drug Administration approved the cancer drug Afinitor (everolimus) on Friday to treat patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis (TS), a rare genetic disorder. This approval was for treatments of SEGA that can not be treated with surgery.

TS causes benign (non-cancerous) tumors to grow in the brain and in other parts of the body including the eyes, lungs, liver, heart, skin and kidneys. TS occurs as a result of genetic mutations that lead to the development of tumors and results in a variety of possible symptoms including learning and developmental disabilities, skin abnormalities, seizures, and lung and kidney disease.

SEGAs are considered a major diagnostic feature of TS and are slow growing tumors, seen in 6 percent to 9 percent of patients. The disease can be fatal for patients who develop complications with tumor growth on the brain. Surgery is used to remove the tumor growths in some patients.

The drug was approved under the FDA’s accelerated approval program. The program allows the FDA to approve a drug to treat serious diseases with an unmet medical need based on an endpoint thought to reasonably predict clinical benefit. The company is required to collect additional long term efficacy and safety data confirming the drug’s benefit. This program provides earlier patient access to promising new or existing drugs while the confirmatory clinical trials are being conducted.

Afinitor was first approved in March 2009 to treat kidney cancer after patients fail treatment with Sutent (sunitinib) or Nexavar (sorafenib).

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FDA approves new indication for Afinitor