Rare Disease Report

Phase 3 Study Testing Everolimus for NETs of GI or Lung Origin Meets Primary Endpoint

MAY 21, 2015
RDR Staff

Novartis has announced their placebo-controlled, Phase 3 study examining the efficacy of everolimus (Afinitor) to treat patients with advanced nonfunctional neuroendocrine tumors (NET) of gastrointestinal (GI) or lung origin met its primary endpoint [significant extension of progression-free survival (PFS)]. While details of the data are not being shared at this time (data to be presented at an upcoming ‘major medical meeting’), Novartis did note that the results would be used to submit the drug for regulatory approval.

Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs said:

 "The results will serve as the basis of planned worldwide regulatory filings for everolimus in these two types of NET, bringing us closer to our goal of offering Afinitor for these patients."

"We look forward to presenting the findings from the RADIANT-4 trial of everolimus, which has the potential to become an important treatment option for patients with advanced nonfunctional GI or lung NET."

Clinical Trial

RADIANT-4 is a Phase 3 prospective, double-blind, randomized, parallel group, placebo-controlled, multicenter study examining the efficacy and safety of everolimus (10 mg daily) plus best supportive care versus placebo plus best supportive care in 302 patients with well differentiated advanced NET of GI or lung origin, and no history or active symptoms of carcinoid syndrome, who had documented disease progression within the previous 6 months. The primary endpoint of RADIANT-4 was PFS. Secondary endpoints included safety, objective response rate and overall survival.


Everolimus is approved for locally advanced, metastatic or unresectable progressive NETs of pancreatic origin. It is also approved for advanced renal cell carcinoma, advanced hormone receptor-positive, HER2-negative breast cancer (advanced HR+ BC), and renal angiomyolipoma and tuberous sclerosis complex (TSC), and TSC plus subependymal giant cell astrocytoma (SEGA).


Novartis drug Afinitor® extended progression-free survival in Phase III trial in advanced gastrointestinal or lung neuroendocrine tumors [press release]. Base, Switzerland: Novartis Pharmaceuticals; May 21, 2015. http://www.novartis.com/newsroom/media-releases/en/2015/1923072.shtml
"Gastric neuroendocrine tumour - low mag" by Nephron - Own work. Licensed under CC BY-SA 3.0 via Wikimedia Commons - https://commons.wikimedia.org/wiki/File:Gastric_neuroendocrine_tumour_-_low_mag.jpg#/media/File:Gastric_neuroendocrine_tumour_-_low_mag.jpg

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