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) was previously approved for the following 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.
On March 26, 2025, Exelixis announced that the U.S. FDA has approved cabozantinib (Cabometyx) for patients with previously treated advanced neuroendocrine tumors.
