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FDA Approval

FDA Approval Alert: New Approval for Afinitor Offers Treatment for Two Rare Diseases

February 26, 2016

A new indication has been granted for the Novartis drug Afinitor® (everolimus) for the treatment of progressive, well-differentiated, nonfunctional gastrointestinal and lung neuroendocrine tumors (NET) that are unresectable, locally advanced, or metastatic.

NET are a rare form of cancer that typically presents with poor prognoses and limited treatment options. NET may be defined as functional or nonfunctional, with nonfunctional NET being characterized by symptoms caused by tumor growth, such as intestinal obstruction, pain and bleeding for gastrointestinal NET, and asthma, chronic obstructive pulmonary disease and pneumonia for lung NET.

The approval of Afinitor, which was granted a priority review by the Food and Drug Administration, was based on efficacy and safety data from the RADIANT-4 study. Afinitor reduced the risk of progression in patients with nonfunctional gastrointestinal or lung NET by more than 50%. Median progression-free survival also increased by 7.1 months in patients treated with Afinitor compared with those taking placebo.

The most common treatment-related, grade 3 or 4 adverse events associated with Afinitor use were infections, diarrhea, stomatitis, fatigue, and hyperglycemia.

Afinitor was previously approved for the treatment of advanced HR+/HER2- breast cancer in combination with exemestane after prior endocrine therapy. This approval makes the drug the only approved therapy for patients with lung NET and the only approved oral therapy for patients with gastrointestinal NET.

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