Cabozantinib Offers New Option for Rare Neuroendocrine Tumors

The FDA has approved the multi-kinase inhibitor cabozantinib for certain adult and pediatric patients with rare neuroendocrine tumors.

The U.S. Food and Drug Administration (FDA) has approved cabozantinib (Cabometyx) for patients 12 years and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated neuroendocrine tumors that are either within the pancreas (pancreatic neuroendocrine tumors, or pNET) or outside the pancreas (extra-pancreatic neuroendocrine tumors, or epNET).

Cabozantinib is a targeted therapy that blocks multiple pathways involved in tumor growth and blood vessel development, and is also approved for certain patients with kidney, liver, and thyroid cancers.

*Slow-growing pancreatic neuroendocrine tumors account for less than 2% of pancreatic cancer cases.*

The approval for pNET and epNET treatment was based on results from the randomized, double-blind, placebo-controlled phase III CABINET study, which enrolled patients with unresectable, locally advanced, or metastatic pNET or epNET that had progressed on prior treatment. Patients in both the pNET and epNET cohorts—which included 99 and 199 patients, respectively—were randomly assigned (2:1) to receive either cabozantinib or placebo.

Compared with their respective placebo arms, cabozantinib reduced the risk of pNET progression by 78% and of epNET progression by 60%.

Half of the patients with pNET who were treated with cabozantinib remained free of progression for at least 13.8, compared with 3.3 months in the pNET placebo arm. In patients with epNET treated with cabozantinib, the corresponding median time to progression was 8.5 months versus 4.2 months in the epNET placebo arm.

Cabozantinib led to tumor responses in 18% of patients with pNET and 5% of patients with epNET, while there were no responses observed in either of the placebo arms.

The recommended dose of cabozantinib is 60 mg orally every day for those weighing 40 kg or more, and 40 mg orally every day for children under 40 kg, until disease progression or unacceptable toxicity.

NETs are rare, slow-growing cancers that arise from hormone-producing cells in the pancreas and other tissues. It has been estimated that NETs affect almost 7 people per 100,000 in the U.S., with the highest incidence rates associated with NETs in the lungs and gastroenteropancreatic sites, and NETs of unknown primary origin.

The FDA rendered its decision on March 26, 2025. Check this resource for updated information on all therapeutics regulated by the FDA.