March 26, 2025--Alameda, CA--(BUSINESS WIRE)
Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) has approved cabozantinib for the treatment of patients who:
1) Have previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNETs) in adults and children aged 12 years and older.
2) Have previously treated, unresectable, locally advanced or metastatic, well-differentiated extrapancreatic neuroendocrine tumors (epNETs) in adults and children aged 12 years and older.
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 disease have a poor prognosis. “Neuroendocrine tumors are highly variable in their characteristics among patients, and few treatment options have demonstrated efficacy improvements in such a heterogeneous population,” said Jennife rChan, MD, Master of Public Health, Research Chair of the CABINET trial, Clinical Director of the Gastrointestinal Cancer Center at the Dana-Farber Cancer Institute, and Director of the Carcinoid and Neuroendocrine Tumor Program. “Encouragingly, in the CABINET trial, cabozantinib significantly delayed disease progression regardless of primary tumor location and grade. This FDA approval marks a significant advance and could potentially establish an important new treatment option for patients, unrestricted by somatostatin receptor expression and functional status.”
This FDA approval (adding a sixth indication for cabozantinib) is based on results from the CABINET trial. CABINET was a pivotal Phase 3 trial evaluating the efficacy of cabozantinib versus placebo in two previously treated cohorts of NET patients (advanced pNET and advanced epNET). The 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) updated its clinical practice guidelines for neuroendocrine and adrenal tumors, including cabozantinib as a Class 1 preferred treatment option for most well-differentiated advanced NETs following specific therapies, and a Class 2A preferred treatment option for other forms of advanced NETs, depending on tumor grade and prior treatment requirements.
“As a company dedicated to improving the standard of care for patients with advanced, refractory cancers, we are proud to bring cabozantinib to patients with previously treated advanced neuroendocrine tumors,” said Dr. Amy Peterson, Executive Vice President and Chief Medical Officer of Product Development and Medical Affairs at Exelixis. “I would like to express my sincere gratitude to the Alliance for Clinical Trials in Oncology that conducted the CABINET trial, the FDA that collaborated with us in reviewing this application, and all the patients and physicians involved in this important study. Looking ahead, we are redoubling our commitment to the neuroendocrine tumor community, and we plan to launch our pivotal STELLAR-311 trial in the first half of 2025, which will examine the efficacy of zanzalintinib against everolimus.”
