FDA Okays Everolimus for Rare Type of Pancreatic Cancer

Alison McCook

May 06, 2011

May 6, 2011 — The US Food and Drug Administration (FDA) has approved everolimus (Afinitor, Novartis) to treat patients with advanced pancreatic neuroendocrine tumors (pNETs).

The drug may be used to treat this rare, slow-growing form of pancreatic cancer if it is metastatic or cannot be removed by surgery. Fewer than 1000 new pNET cases occur in the United States each year.

No new drug has been approved for this group of patients in the United States for nearly 30 years.

"Patients with this cancer have few effective treatment options," Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA's 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 approval is based on results from a clinical trial, previously reported by Medscape Medical News, of 410 patients with metastatic or locally advanced disease. Those that received everolimus experienced a median of 11 months before tumor growth compared with 4.6 months in participants who were given a placebo. Patients taking everolimus experienced a 65% lower risk for cancer progression than those receiving placebo (hazard ratio, 0.35 [95% confidence interval, 0.27 - 0.45]; P < .001), according to data released by Novartis.

Most commonly reported adverse effects among patients receiving everolimus included stomatitis, rash, diarrhea, fatigue, edema, abdominal pain, nausea, fever, and headache.

Everolimus, which targets the mammalian target of rapamycin (mTOR), has already been approved for patients with advanced renal cell carcinoma in whom treatment with sunitinib (Sutent, Pfizer) or sorafenib (Nexavar, Bayer Health Care) has failed. It is also prescribed for patients with subependymal giant cell astrocytoma that is associated with tuberous sclerosis and cannot be treated surgically. Everolimus is also marketed under another trade name, Zortress, under which it is used to prevent organ rejection in some adult kidney transplant recipients.

Novartis originally sought approval to market everolimus for other neuroendocrine tumors, then amended its application to focus only on advanced pNETs, "based on feedback from the FDA," the company said in a statement.

"The FDA approval of Afinitor represents an important step forward for patients with advanced pancreatic NET," James Yao, MD, associate professor of medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, said in a statement from Novartis. "Patients will now have access to a treatment that has been shown to significantly delay tumor growth and reduce the risk of disease progression."

More information is available on the FDA Web site.

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