The investigational drug cabozantinib (CABOMETYX®) has significantly improved outcomes for those living with advanced neuroendocrine tumors (NETs), according to the latest results from the Phase 3 CABINET trial.
The results, reported in September in The New England Journal of Medicine, demonstrate that cabozantinib extended progression-free survival (PFS) for both pancreatic and extra-pancreatic NETs, providing a much-needed option for patients whose cancer has progressed after previous treatments such as PRRT or targeted therapy.
Specifically, for study participants with extra-pancreatic NETs, cabozantinib extended PFS to 8.4 months, compared to just 3.9 months for those receiving a placebo. For those with pancreatic NETs, PFS increased to 13.8 months with cabozantinib compared to 4.4 months with placebo.
While side effects such as high blood pressure, fatigue, diarrhea, and blood clots were common, they were consistent with the drug’s known safety profile. No new safety concerns were identified in the trial, making cabozantinib a promising option for patients with limited treatment choices.
These results represent a significant breakthrough in the fight against NETs, where treatment options have been historically limited, especially for patients whose tumors have progressed after previous therapies.
NETRF has played an important role in advancing research on cabozantinib. In 2018, NETRF funded a pilot project led by Dr. Jennifer Chan, the study chair for the CABINET trial, to explore biomarkers that might predict patient responses to cabozantinib.
In August, Exelixis announced that the U.S. Food and Drug Administration accepted its supplemental New Drug Application (sNDA) for cabozantinib (CABOMETYX®) to treat adults with advanced pancreatic and extra-pancreatic NETs.
The FDA has set a target action date of April 3, 2025, by which time it will complete its review and decide whether to approve cabozantinib for this new use. This sNDA submission marks a crucial step in expanding treatment options for NET patients.