New Indication Approved for Nonfunctional GI and Lung Neuroendocrine Tumors

March 3, 2016
Bryant Furlow

The FDA has approved everolimus (Afinitor®, Novartis) for use in the treatment of adult patients who have been diagnosed with locally-advanced or metastatic progressive, well-differentiated, nonfunctional neuroendocrine tumors (NET) primary to the GI tract or lung.

The US Food and Drug Administration (FDA) has approved everolimus (Afinitor®, Novartis) for use in the treatment of adult patients who have been diagnosed with locally-advanced or metastatic progressive, well-differentiated, nonfunctional neuroendocrine tumors (NET) primary to the gastrointestinal (GI) tract or lung.

NET are a rare cancer of the neuroendocrine cells, usually found in the pancreas, lungs, or GI tract. Functional NET cause hormone oversecretion and nonfunctional NET symptoms include obstruction, bleeding, and pain caused by the growth of the tumor.

“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 Novartis Oncology President Bruno Strigini, in a press release.  

The approval was based on safety and efficacy findings from the prospective phase III, randomized, placebo-controlled RAD001 In Advanced Neuroendocrine Tumors (RADIANT)-4 clinical trial, which demonstrated that, compared to placebo, everolimus reduced risk of progression by 52% (hazard ratio [HR] = 0.48; 95% CI: 0.35-0.67; P < .001). Trial data further demonstrated a progression-free survival (PFS) of 11.0 months by central review, compared to 3.9 months for patients in the study’s control group, who received best supportive care plus placebo.

Patients with NET of pancreatic origin were excluded from the RADIANT-4 trial.

The safety profile for everolimus was “consistent with what has been observed in previous studies,” the company announced. The most common everolimus-related grade 3/4 adverse events included infections (11% vs 2% in the placebo group); diarrhea (9% vs 2%); stomatitis (9% vs 0%); fatigue (5% vs 1%); and hyperglycemia (5% vs 0%), according to the Novartis announcement.

The most common treatment-related adverse events of all grades, occurring in 30% or more of patients on everolimus, included stomatitis (63%); infections (58%); diarrhea (41%); peripheral edema (39%); fatigue (37%); and rash (30%), the company reported.

The recommended dose for everolimus is 10 mg orally, taken once daily.