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FDA Approves Everolimus for GI, Lung NETs

Silas Inman @silasinman
Published: Friday, Feb 26, 2016

Dr. James C. Yao

James C. Yao, MD

The FDA has approved everolimus (Afinitor) for the treatment of adult patients with progressive, well-differentiated non-functional, locally advanced or metastatic gastrointestinal (GI) or lung neuroendocrine tumors (NET), based on findings from the phase III RADIANT-4 trial.1

Data from a subgroup analysis looking specifically at GI NETs further illuminated the benefit in this population.2 In the GI subgroup, median PFS was 13.1 months with everolimus versus 5.4 months with placebo (HR, 0.56; 95% CI, 0.37-0.84). In the midgut subgroup, median PFS was 17.3 months with everolimus versus 10.9 months with placebo (HR, 0.71; 95% CI, 0.40-1.26). Among patients with non-midgut NETS, median PFS was 8.1 months versus 1.9 months, respectively (HR, 0.27; 95% CI, 0.15-0.51).

“Everolimus is an effective and new exciting treatment option in a disease where we’ve had very few treatment options to date,” lead study author Simron Singh, MD, a medical oncologist at Sunnybrook’s Odette Cancer Centre in Toronto, Canada, said when presenting the data ahead of the 2016 Gastrointestinal Cancers Symposium. “Regardless of prior treatment, the patients appeared to benefit from everolimus across the board."

Adverse events (AEs) were consistent with the known safety profile for everolimus. Drug-related AEs were mostly grades 1 and 2 stomatitis, diarrhea, fatigue, infections, rash, and peripheral edema. The most common grades 3/4 AEs among everolimus-treated patients were stomatitis (9%), diarrhea (7%), and infections (7%). Grade 3/4 adverse events were uncommon in the placebo arm.

Patients received everolimus for a median of 40.4 weeks. The main reasons for treatment discontinuations were disease progression and AEs. Disease progression occurred in 37% of patients treated with everolimus versus 72% of those in the placebo arm. AEs accounted for discontinuations in 29% and 7% for everolimus and placebo, respectively.

"Afinitor is the first treatment approved for progressive, nonfunctional NET of lung origin, and one of very few options available for progressive, nonfunctional GI NET, representing a shift in the treatment paradigm for these cancers," said Bruno Strigini, President, Novartis Oncology, the company developing the medication. "We are proud of our Afinitor development program, which has translated to meaningful benefits for patients with several different cancers and rare diseases."

In addition to NETs, everolimus is approved for several indications, including HR-positive, HER2-negative breast cancer in combination with exemestane and for patients with advanced renal cell carcinoma following progression on frontline sunitinib or sorafenib. The agent also holds approvals for subependymal giant cell astrocytoma. 


  1. Yao J, Fazio N, Singh S, et al. Everolimus in advanced nonfunctional neuroendocrine tumors (NET) of lung or gastrointestinal (GI) origin: efficacy and safety results from the placebo-controlled, double-blind, multicenter, Phase III RADIANT-4 study. Presented at: 2015 European Cancer Congress; September 25-29; Vienna, Austria. Abstract LBA5.
  2. Singh S, Carnaghi C, Buzzoni R, et al. Efficacy and safety of everolimus in advanced, progressive, nonfunctional neuroendocrine tumors (NET) of the gastrointestinal (GI) tract and unknown primary: A subgroup analysis of the phase III RADIANT-4 trial. J Clin Oncol. 2016;34 (suppl 4S; abstr 315).


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