FDA Approves Afinitor for Pancreatic Cancer

Afinitor (everolimus), a targeted anticancer drug that has been on the market since 2009, is now approved for the treatment of pancreatic neuroendocrine tumors (pNET) in patients with unresectable, locally advanced, or metastatic disease.

The FDA’s approval of the expanded indication, announced Friday, marks the first new treatment approved for advanced pNET in nearly 30 years, according to Novartis, which manufactures the drug.

The company said approximately 60% of patients with pNET, a relatively uncommon type of cancer, exhibit advanced disease at the time of diagnosis and that the 5-year survival rate for these patients is 27%.

"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 press release. “Afinitor has demonstrated the ability to slow the growth and spread of neuroendocrine tumors of the pancreas."

The new indication was approved on the strength of the phase III RADIANT-3 trial in which 410 patients were randomly selected to receive either Afinitor or a placebo. For those in the Afinitor arm, the median progression-free survival time was 11 months, compared with 4.6 months for those who received the placebo.

Afinitor, which is administered orally, also is approved for the treatment of patients with advanced renal cell carcinoma after the failure of Sutent (sunitinib) or Nexavar (sorafenib), and for patients with subependymal giant cell astrocytoma associated with tuberous sclerosis.

Novartis said marketing applications are pending with the European Medicines Agency and with other regulatory agencies worldwide to use the drug in patients with advanced neuroendocrine tumors of gastrointestinal, lung, or pancreatic origin.

The FDA approval comes less than a month after the agency’s Oncologic Drugs Advisory Committee voted unanimously to support it. In a separate vote, the panel also backed the use of Sutent for unresectable pNET, but members were divided. The FDA has not yet made a final decision on an expanded Sutent indication.

Additional resources:

• FDA News Release
• Novartis Media Release
• Prescribing Information
• ODAC Panel Background