The Food and Drug Administration has approved the use of everolimus (Afinitor, Novartis) to treat patients with progressive neuroendocrine tumors located in the pancreas (PNET) that cannot be removed by surgery or that have metastasized).

The FDA's action follows the recommendation by the Oncologic Drugs Advisory Committee on April 12 that the indication should be approved.

Such tumors are slow-growing and very rare, with fewer than 1,000 new cases in the United States each year.

"Patients with this cancer have few effective treatment options," Richard Pazdur, MD, Director of the FDA's Office of Oncology Drug Products in the Center for Drug Evaluation and Research, said in a statement. "Afinitor has demonstrated the ability to slow the growth and spread of neuroendocrine tumors of the pancreas."

The safety and effectiveness of Afinitor were established a clinical trial in 410 patients with metastatic or locally advanced disease. Patients in the study were selected to receive Afinitor or placebo, and the trial was designed to measure the length of time a patient lived before their disease spread or worsened.

In patients treated with Afinitor, the median length of time they lived without the cancer spreading or worsening was 11 months vs 4.6 months in patients who received placebo. Patients who received placebo were able to receive Afinitor if their disease worsened.

In patients treated with Afinitor for PNET, the most commonly reported side effects included stomatitis, rash, diarrhea, fatigue, edema, abdominal pain, nausea, fever, and headache.