The FDA has approved cabozantinib (Cabometyx) for the treatment of adult and pediatric patients aged 12 years or older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET) and well-differentiated extra-pancreatic neuroendocrine tumors (epNET).
The regulatory decision is supported by findings from 2 cohorts of the phase 3 CABINET trial (NCT03375320). In the pNET cohort (n = 99), the agent led to 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) with placebo (HR, 0.22; 95% CI, 0.12-0.41; _P_ < .0001). The overall response rate with cabozantinib was 18% (95% CI, 10%-30%) vs 0% (95% CI, 0%-11%) with placebo. Overall survival (OS) data were not mature (HR, 1.01).
In the epNET cohort (n = 199), cabozantinib led to a median PFS of 8.5 months (95% CI, 6.8-12.5) vs 4.2 months (95% CI, 3.0-5.7) with placebo (HR, 0.40; 95% CI, 0.26-0.61; _P_ < .0001). The respective ORRs were 5% (95% CI, 2.2%-11%) and 0% (95% CI, 0%-5%). OS data were not mature in this cohort either (HR, 1.01).
The toxicity profile of cabozantinib aligned with the approved product label, according to the regulatory agency.
### Reference
FDA approves cabozantinib for adults and pediatric patients 12 years of age and older with pNET and epNET. FDA. March 26, 2025. Accessed March 26, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-cabozantinib-adults-and-pediatric-patients-12-years-age-and-older-pnet-and-epnet