The US Food and Drug Administration (FDA) has approved everolimus, made by Novartis and marketed as Afinitor, for the treatment of advanced kidney cancer, or renal cell carcinoma.

The approval comes after early data from a phase III clinical trial showed Afinitor delayed the growth and spread of kidney tumors by about 5 months, compared to a 2-month delay experienced by patients who were not taking the drug.

The FDA approved the use of Afinitor for patients whose advanced renal cell cancer is no longer responding to Sutent (sunitinib) or Nexavar (sorafenib), two targeted therapies commonly used to treat the disease. Like Sutent and Nexavar, Afinitor is a kinase inhibitor. But where those drugs act on a number of regions, it targets one protein, interrupting tumor cell division and blood vessel growth.

Targeted drugs are especially important to kidney cancer patients because other treatments such as chemotherapy have not been shown to be very effective. For more information, see Kidney Cancer: Targeted Therapies.

This drug does not cure kidney cancer, and only 2% of patients had their tumors shrink significantly. But Afinitor does slow tumor growth for a time. After 10 months of treatment with Afinitor, approximately 75% of patients' tumors had begun growing again.

"Afinitor provides an option for patients with advanced renal cell cancer after failure of treatment with the cancer therapies sunitinib or sorafenib," said Robert Justice, MD, Director, Division of Drug Oncology Products in the FDA's Center for Drug Evaluation and Research. "Targeted cancer therapies like Afinitor have increased the number of months patients can live without the tumor progressing."

Commonly reported side effects include mouth sores, weakness, diarrhea, poor appetite, fluid buildup in the arms and legs, nausea, vomiting, rash, fever, and shortness of breath.

Novartis plans to test Afinitor for use in the treatment of lymphoma, breast, stomach, lung, and other cancers.

"With this approval, we can now offer patients a targeted therapy proven to fulfill an important unmet need in the treatment of advanced kidney cancer," said David Epstein, President and CEO, Novartis Oncology, Novartis Molecular Diagnostics. "We continue to study Afinitor in kidney cancer, and through a broad clinical program to explore its potential in many other tumor types."

Reviewed by: Members of the ACS Medical Content Staff