Phase 3 Trial Leads to FDA Approval of Cabometyx in Neuroendocrine Tumors

The U.S. Food and Drug Administration (FDA) has approved Cabometyx (cabozantinib) for some patients 12 years of age and older with previously treated neuroendocrine tumors. The approval was based on the findings of the phase 3 CABINET trial.

The trial, Dr. Jennifer Chan told CURE, offered “clear evidence that the drug can slow disease progression and growth.” Chan, clinical director of the Gastrointestinal Cancer Center and director of the Program in Carcinoid and Neuroendocrine Tumors at Dana-Farber Cancer Institute in Boston, led the trial.

The regulatory agency approved Cabometyx for the treatment of adult and pediatric patients who are 12 years old and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET) and well-differentiated extra-pancreatic neuroendocrine tumors (epNET).

In light of the approval, Chan spoke with CURE about the CABINET trial and its findings.

Transcript:

The study that led to the approval was a phase 3 trial conducted in patients with advanced neuroendocrine tumors, all of whom had disease that had progressed after at least one prior FDA-approved line of therapy. The trial had two independent cohorts: one group of patients with pancreatic neuroendocrine tumors and the other with neuroendocrine tumors originating outside the pancreas (extra-pancreatic neuroendocrine tumors). It was a randomized trial comparing Cabometyx, starting at a dose of 60 milligrams a day, to placebo. The primary outcome of the study was to assess progression-free survival.

What the researchers observed in both groups was that Cabometyx significantly improved progression-free survival compared with placebo. Specifically, there was a 62% reduction in the risk of disease progression or death in patients with extra-pancreatic neuroendocrine tumors, and a 77% reduction in the risk of disease progression or death in those with pancreatic neuroendocrine tumors. This provided clear evidence that Cabometyx can slow disease progression and control tumor growth. Based on this demonstrated effectiveness, the drug was approved, adding another treatment option for patients.

It’s important to note that there are multiple therapies that can be considered for patients with advanced NET. The good news is that there is another standard therapy for patients and their providers to consider.We can choose which option to use based on each person's individual situation and the specific characteristics of their disease.

Transcript has been edited for clarity and conciseness.

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.