Dr Chan on the FDA Approval of Cabozantinib for Pancreatic and Extra-Pancreatic Neuroendocrine Tumors

"This approval really means that we have a new standard option to offer to patients with neuroendocrine tumors who've had at least 1 other line of therapy and whose disease is progressing and needs additional therapy to control growth."

Jennifer Chan, MD, MPH, an associate professor of medicine at Harvard Medical School and clinical director of the Gastrointestinal Cancer Center at Dana-Farber Cancer Institute, discussed the clinical relevance of the FDA approval of cabozantinib (Cabometyx) for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNETs) and extra-pancreatic neuroendocrine tumors (pNETS).

The regulatory decision on March 26, 2025, was supported by data from the phase 3 CABINET trial (NCT03375320), which showed that among patients in the pNET cohort (n = 99), those treated with cabozantinib experienced a median progression-free survival (PFS_ of 13.8 months (95% CI, 8.9-17.0) vs 3.3 months (95% CI, 2.8-5.7) for those given placebo (HR, 0.22; 95% CI, 0.12,-0.41; P < .0001). In the epNET cohort, the median PFS was 8.5 months (95% CI, 6.8- 12.5) in the cabozantinib arm vs 4.2 months (95% CI, 3.0-5.7) in the placebo arm (HR, 0.40; 95% CI, 0.26- 0.61; P < .0001).

Chan noted that the approval of cabozantinib represents a key advancement in the treatment landscape for patients with pNETs and epNETs. Chan highlighted the importance of this milestone, particularly given the limited treatment options available for this patient population. The broad label indication is notable, encompassing patients with both functional and nonfunctional tumors, as well as those with somatostatin receptor–positive and –negative disease.

Chan concluded by noting that cabozantinib offers a new, broadly applicable therapeutic option for patients with progressive NETs, addressing a longstanding unmet need in this field. As treatment options for NETs continue to expand, the integration of cabozantinib into clinical practice will provide oncologists with an effective tool for disease management in a heterogeneous and challenging patient population, she said.