FDA Approves Cabometyx (Cabozantinib) for the Treatment of Previously Treated Patients with Advanced

On March 26, 2025, Exelixis, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Cabometyx (cabozantinib) for the treatment of two patient populations:

Adult and pediatric patients 12 years of age and older with previously treated, unresectable locally advanced or metastatic well-differentiated pancreatic neuroendocrine tumors (pNET);

Adult and pediatric patients 12 years of age and older with previously treated, unresectable locally advanced or metastatic well-differentiated extrapancreatic neuroendocrine tumors (epNET).

Neuroendocrine tumors (NETs) are a heterogeneous group of tumors arising from neuroendocrine cells in the digestive tract, lung, pancreas, and other organs. Most patients with advanced disease have a poor prognosis.

This FDA approval marks the sixth approval for cabozantinib, based on results from the Phase 3 pivotal CABINET trial. The trial evaluated cabozantinib versus placebo in two cohorts of previously treated patients with advanced neuroendocrine tumors (pancreatic and extrapancreatic). Final progression-free survival results were presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in The New England Journal of Medicine.

In January 2025, the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology: Neuroendocrine and Adrenal Tumors were updated to list cabozantinib as a Category 1 preferred regimen for most previously treated patients with well-differentiated advanced neuroendocrine tumors, and a Category 2A preferred regimen for other types of advanced neuroendocrine tumors (depending on tumor grade and prior therapy requirements).

Previously Approved Indications for Cabometyx (Cabozantinib)

April 26, 2016

Exelixis announced FDA approval of Cabometyx for patients with advanced renal cell carcinoma (RCC).

December 19, 2017

Exelixis announced FDA approval of Cabometyx tablets for the first-line treatment of patients with advanced renal cell carcinoma.

January 14, 2019

Exelixis announced FDA approval of Cabometyx tablets for patients with previously treated hepatocellular carcinoma (HCC).

January 22, 2021

FDA approved Opdivo (nivolumab) in combination with Cabometyx as first-line treatment for patients with advanced renal cell carcinoma.

September 17, 2021

Exelixis announced FDA approval of Cabometyx for patients with previously treated radioactive iodine-refractory differentiated thyroid cancer.

March 26, 2025

Exelixis announced FDA approval of Cabometyx for patients with previously treated advanced neuroendocrine tumors.