Rare Disease Report

FDA Approves Afinitor Tablets for Neuroendocrine Tumors

FEBRUARY 26, 2016
James Radke, PhD
The FDA has approved Afinitor (everolimus) tablets for the treatment of adult patients with progressive, well-differentiated, nonfunctional neuroendocrine tumors of gastrointestinal or lung origin that are unresectable, locally advanced or metastatic.
"Afinitor is the first treatment approved for progressive, nonfunctional neuroendocrine tumors of lung origin, and one of very few options available for progressive, nonfunctional gastrointestinal neuroendocrine tumors, representing a shift in the treatment paradigm for these cancers," said Bruno Strigini, President, Novartis Oncology.
Neuroendocrine tumors are a rare type of cancer that originate in neuroendocrine cells throughout the body, and are most often found in the gastrointestinal tract, lungs or pancreas. These tumors can be defined as functional or nonfunctional. Functional neuroendocrine tumors are characterized by symptoms caused by the oversecretion of hormones and other substances. Nonfunctional neuroendocrine tumors may be characterized by symptoms caused by tumor growth, such as intestinal obstruction, pain and bleeding for gastrointestinal neuroendocrine tumors, and asthma, chronic obstructive pulmonary disease and pneumonia for lung neuroendocrine tumors.
Most neuroendocrine tumors (>70%) are nonfunctional tumors.

Pivotal Trial

RADIANT-4 (RAD001 In Advanced Neuroendocrine Tumors) is a Phase 3 prospective, double-blind, randomized, parallel group, placebo-controlled, multicenter study that compared Afinitor plus best supportive care vs placebo plus best supportive care in 302 patients with unresectable, progressive, well-differentiated, nonfunctional, locally advanced or metastatic neuroendocrine tumor of gastrointestinal (excluding pancreatic) or lung origin. The major efficacy outcome measure of RADIANT-4 was progressive free survival (PFS) based on independent radiological assessment evaluated by Response Evaluation Criteria in Solid Tumors.
Patients were randomized 2:1 to receive daily Afinitor 10 mg or daily placebo orally. All patients received BSC during treatment, which excluded somatostatin analogues (SSAs). Patients had low or intermediate grade histology, no history or active symptoms of carcinoid syndrome, had documented disease progression within the previous 6 months and were required to have ceased treatment with SSAs for 4 weeks before study entry.
Afinitor is also approved for treating:
  • postmenopausal women with advanced hormone receptor-positive, HER2-negative breast cancer (advanced HR+ BC) in combination with exemestane after failure of treatment with letrozole or anastrozole.
  • adults with progressive neuroendocrine tumors of pancreatic origin
  • adults with advanced renal cell carcinoma (RCC) after failure of treatment with sunitinib or sorafenib
  • adults with renal angiomyolipoma and tuberous sclerosis complex (TSC), not requiring immediate surgery.

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