National Cancer Institute NCI Cancer Bulletin: A Trusted Source for Cancer Research News
May 17, 2011 • Volume 8 / Number 10

FDA Update

FDA Approves Drug to Treat Pancreatic Neuroendocrine Tumors

The Food and Drug Administration (FDA) has approved everolimus (Afinitor) for the treatment of pancreatic neuroendocrine tumors (PNET) that cannot be surgically removed or that have metastasized. This is only the second drug approved to treat this rare form of pancreatic cancer, which is diagnosed in fewer than 1,000 patients annually in the United States.

The FDA’s approval was based on the results of a recent randomized trial involving patients with advanced PNET. Participants treated with everolimus lived an average of 6.4 months longer without disease progression than those who took a placebo.

Everolimus is also approved for treating renal cell carcinoma and subependymal giant cell astrocytoma, a type of benign brain tumor. The drug is designed to prevent cells from proliferating and tumor blood vessels from forming by inhibiting the mammalian target of rapamycin (mTOR) signaling pathway. Common side effects include rash, diarrhea, fatigue, and abdominal pain; serious complications, including kidney failure, have also been reported.

Everolimus is one of two targeted therapies that the FDA’s Oncologic Drugs Advisory Committee recently recommended for approval to treat PNET.

Further reading: Targeted Therapies May Be Effective Against Rare Pancreatic Cancer