Neuroendocrine tumors that start in the pancreas tend to be more aggressive and have a poor prognosis.
The FDA has approved a supplemental application for Cabometyx (cabozantinib) to treat adult and pediatric patients 12 years of age and older with pancreatic neuroendocrine tumors. It is indicated for those with previously treated, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET), as well as locally advanced or metastatic, well-differentiated extra-pancreatic NET (epNET).
Neuroendocrine tumors develop in the neuroendocrine cells of the digestive tract and other organs, such as the lung and pancreas. In 2024, the estimated prevalence of NET in the United States was more than 380,000 people. It is estimated that 161,000 to 192,000 people are living with unresectable, locally advanced or metastatic NET. Neuroendocrine tumors that start in the pancreas tend to be more aggressive, with a five-year survival rate of only 23% for advanced disease.
Jennifer Chan, M.D.
“The characteristics of NET vary widely from patient to patient, and very few treatment options have demonstrated the ability to improve outcomes across such a heterogeneous population,” Jennifer Chan, M.D., clinical director of the Gastrointestinal Cancer Center and director of the Program in Carcinoid and Neuroendocrine Tumors at Dana-Farber Cancer Institute, said in a news release.
Cabometyx is already available to treat patients with advanced renal cell carcinoma (RCC), hepatocellular carcinoma (HCC) and metastatic differentiated thyroid cancer (DTC). Developed by Exelixis, Cabometyx targets tumor cells in which MET, AXL, and VEGF are overexpressed. These receptors are involved in tumor angiogenesis, invasiveness, metastasis, and immunomodulation of the tumor microenvironment.
In 2024, Cabometyx generated U.S. revenue of $1.81 billion, and company executives indicated in its year-end earnings call that there has been about 11% growth year over year in demand and new prescription starts and revenue for Cabometyx.
The cost for Cabometyx 20 mg oral tablet is around $27,468 for a supply of 30 tablets, according to Drugs.com. Commercially insured patients may pay a $0 a month copay. The terms of the copay program indicate that Exelixis may reduce or eliminate the copay assistance if a patient’s insurance plan uses an accumulator or maximizer program. The company also offers a free, 30-day trial program.
The approval for the pancreatic neuroendocrine tumors indication is based on data from CABINET, a phase 3 pivotal trial. The trial compared Cabometyx with placebo in two cohorts of patients with previously treated NET: advanced pNET and advanced epNET.
Final progression-free survival results were presented at the 2024 European Society for Medical Oncology Congress in September 2024 and published in The New England Journal of Medicine. In the pNET cohort of 95 patients at a median follow-up of 13.8 months, the median progression-free survival was 13.8 months for Cabometyx and 4.4 months for placebo. In the epNET cohort of 203 patients at a median follow-up of 10.2 months, the median progression-free survival was 8.4 months for Cabometyx and 3.9 months for placebo.
No new safety signals were identified, but a majority of patients treated with Cabometyx required dose modifications or reductions to manage adverse events.
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