On March 26, 2025, the U.S. Food and Drug Administration (FDA) approved cabozantinib (Cabometyx) as a treatment for adults and children aged 12 and older with certain types of neuroendocrine tumors. These tumors include well-differentiated pancreatic neuroendocrine tumors that cannot be surgically removed or have spread to other parts of the body, as well as extra-pancreatic neuroendocrine tumors, which occur outside the pancreas.
Cabozantinib, developed by the pharmaceutical company Exelixis, works by targeting and blocking signals that promote tumor growth and spreading. This approval was based on clinical trial results that showed the drug significantly improved how long patients lived without their disease worsening, compared to those receiving a placebo (a pill with no active medicine).
In addition to its primary role in treating neuroendocrine tumors, cabozantinib has been previously approved for other cancer types, including advanced kidney cancer and hepatocellular carcinoma (a type of liver cancer). The most common side effect associated with cabozantinib is high blood pressure, which may require dosage adjustments. Other potential side effects include fatigue, diarrhea and liver problems.
This new treatment option provides hope for patients with advanced neuroendocrine tumors, offering a more effective alternative when previous treatments have not worked.
Disclaimer: This article is for informational purposes only. Always consult your healthcare provider before starting any new treatment.