On March 26, 2025, Exelixis announced that the U.S. Food and Drug Administration (FDA) has approved cabozantinib (Cabometyx) for the treatment of two patient groups:
1) Adult and pediatric patients aged 12 years and older with previously treated, unresectable locally advanced or metastatic well-differentiated pancreatic neuroendocrine tumors (pNETs);
2) Adult and pediatric patients aged 12 years and older with previously treated, unresectable locally advanced or metastatic well-differentiated extrapancreatic neuroendocrine tumors (epNETs).
Neuroendocrine tumors (NETs) are a heterogeneous group of tumors originating from neuroendocrine cells in the digestive tract and other organs such as the lungs and pancreas. Most patients with advanced NETs have a poor prognosis.
This FDA approval is the sixth such approval for cabozantinib, based on the results of the CABINET trial. This is a pivotal phase 3 trial evaluating the efficacy of cabozantinib versus placebo in two groups of previously treated neuroendocrine tumor patients (advanced pancreatic neuroendocrine tumors and advanced extrapancreatic neuroendocrine tumors). Final progression-free survival results were presented at the 2024 European Society for Medical Oncology Congress and published in the *New England Journal of Medicine*.
In January 2025, the *National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines for Oncology: Neuroendocrine and Adrenal Tumors* were updated, listing cabozantinib as a Category 1 preferred treatment for most well-differentiated advanced neuroendocrine tumors that have received specific therapies, and a Category 2A preferred treatment for other types of advanced neuroendocrine tumors (depending on tumor grade and different requirements of prior treatment).
Cabozantinib (Cabometyx) Previously Approved Indications
April 26, 2016
Exelixis announced that the FDA approved cabozantinib (Cabometyx) for patients with advanced renal cell carcinoma.
December 19, 2017
Exelixis announced that the FDA approved cabozantinib (Cabometyx) tablets for the treatment of previously untreated patients with advanced renal cell carcinoma.
January 14, 2019
Exelixis announced that the FDA approved cabozantinib (Cabometyx) tablets for the treatment of previously treated patients with hepatocellular carcinoma.
January 22, 2021
The FDA approved Opdivo (nivolumab) in combination with cabozantinib (Cabometyx) as a first-line treatment for patients with advanced renal cell carcinoma.
September 17, 2021
Exelixis announced that the FDA approved cabozantinib (Cabometyx) for the treatment of previously treated patients with radioactive iodine-refractory differentiated thyroid carcinoma.
March 26, 2025
Exelixis announced that the U.S. FDA has approved cabozantinib (Cabometyx) for patients with previously treated advanced neuroendocrine tumors.
