Cabozantinib gains FDA approval for treating advanced neuroendocrine tumors

Cabozantinib, an oral tyrosine kinase inhibitor, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with previously treated advanced neuroendocrine tumors (NETs). This represents a new standard of care therapy for this patient population.

The FDA approval was based on results from the CABINET study, a phase 3 pivotal trial evaluating cabozantinib compared with placebo in two groups of patients with previously treated NETs: advanced pancreatic NETs and advanced extra-pancreatic NETs. The study was led by Jennifer Chan, MD, MPH, clinical director of the gastrointestinal cancer center and director of the program in carcinoid and neuroendocrine tumors at Dana-Farber Cancer Institute.

Patients with neuroendocrine tumors often face a difficult journey. Despite advances in recent years, there has remained a critical need for new and effective therapies for patients whose cancer has grown or spread. Cabozantinib significantly improved outcomes in this patient population and this FDA approval provides new hope."

Jennifer Chan, MD, MPH, clinical director of the gastrointestinal cancer center and director of the program in carcinoid and neuroendocrine tumors at Dana-Farber Cancer Institute

Cabozantinib works by targeting multiple pathways involved in tumor growth and angiogenesis. Patients with NETs treated with the drug survived significantly longer with no worsening of their disease compared to patients who received a placebo. Side effects of cabozantinib were like those found in other studies of the drug. These include hypertension, fatigue, and diarrhea.

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. Based on improved efficacy at interim analysis, the trial was stopped early and unblinded in August 2023.

More than 12,000 people in the United States are diagnosed with a NET each year. The tumors begin in neuroendocrine cells – which have characteristics of nerve and hormone-producing cells – and can arise in multiple sites in the body, most often in the gastrointestinal tract, lungs, and pancreas. The number of people diagnosed with NETs has been increasing in recent decades. Treatments may include surgery, molecular targeted therapy, peptide receptor radionuclide therapy, chemotherapy, or other local treatment approaches depending on the location and stage of the cancer. For patients whose cancer continues to grow and spread after these treatments, better options are urgently needed.

The CABINET trial was sponsored by the NCI, part of the National Institutes of Health (U10CA180821, U10CA180882), and was led and conducted by the NCI-funded Alliance for Clinical Trials in Oncology with participation from the NCTN as part of Exelixis' collaboration with the NCI's Cancer Therapy Evaluation Program (NCI-CTEP); https://acknowledgments.alliancefound.org.