Cabometyx wins FDA approval for treating certain pancreatic cancers
Medication now OK’d for patients 12 and older with neuroendocrine tumors

The U.S. Food and Drug Administration (FDA) has granted its approval to the oral medication Cabometyx (cabozantinib) as a treatment for certain types of pancreatic cancer.
The approval specifically covers the use of Cabometyx for patients ages 12 and older who have previously treated, unresectable — meaning the cancer cannot be surgically removed — locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors. The therapy is also now cleared for treating neuroendocrine tumors outside of the pancreas.
Exelixis, which owns the rights to develop and commercialize Cabometyx in the U.S., said in a company press release that it “is prepared to immediately support these new indications.” Cabometyx was already FDA-approved to treat some types of liver, kidney, and thyroid cancers.
“As a company committed to improving the standard of care for people living with advanced, difficult-to-treat cancers, we are proud to bring Cabometyx to patients with previously treated advanced neuroendocrine tumors,” said Amy Peterson, MD, chief medical officer and executive vice president of product development and medical affairs at Exelixis.
Oral therapy already approved in US to treat liver, kidney, thyroid cancers
Neuroendocrine tumors, known as NETs, are a specific type of cancer. These tumors arise from the uncontrolled growth of cells that combine features of hormone-producing endocrine cells and nerve cells. The cells are found throughout the body, particularly in the gastrointestinal tract, pancreas, and lungs. But pancreatic NETs account for fewer than 10% of pancreatic tumors.
To date, Cabometyx is authorized for various indications in more than 60 countries worldwide, according to Exelixis. Rights to the therapy in Japan are owned by Takeda Pharmaceutical, while rights in all other countries are owned by Ipsen Pharma.
Cabometyx is a targeted therapy that blocks multiple tyrosine kinases, which are signaling proteins involved in tumor growth, tumor spread, and angiogenesis, or the formation of new blood vessels.
According to Jennifer Chan, MD, of Dana-Farber Cancer Institute, “this FDA approval marks a meaningful advancement, which may establish an important new treatment option for patients, without limitations based on” other tumor factors.
“The characteristics of NET vary widely from patient to patient, and very few treatment options have demonstrated the ability to improve outcomes across such a heterogeneous population,” said Chan, director of the program in carcinoid and neuroendocrine tumors at Dana-Farber.
Approval of Cabometyx for pancreatic and nonpancreatic NETs was based on data from a Phase 3 clinical trial called CABINET (NCT03375320). The study enrolled 95 people with pancreatic NETs and 203 with nonpancreatic NETs, all of whom had received at least one prior line of treatment. Participants were randomly assigned to take Cabometyx at a dose of 60 mg daily or a placebo.
The main goal of the study was to measure how long patients lived without their cancer getting worse. Secondary goals included how well the treatment shrank tumors, overall survival, and safety.
New Cabometyx approval based on positive results of Phase 3 CABINET trial
The trial results showed that, in pancreatic NET, those treated with Cabometyx went an average of 13.8 months, or a little longer than a year, without their cancer worsening. That compared with a span of 4.4 months for the patients who received the placebo. In nonpancreatic NET, Cabometyx patients experienced an average of 8.4 months of progression-free survival, compared with 3.9 months with the placebo.
In 19% of patients with pancreatic NET and 5% of those with nonpancreatic NET, Cabometyx led to a reduction in tumor size. Meanwhile, none of the patients given the placebo experienced a reduction in tumor size.
“It was encouraging to see that [Cabometyx] resulted in significant delays in disease progression in the CABINET trial — regardless of primary tumor site and grade,” said Chan, who served as study chair for the CABINET trial.
Safety data from the trial were consistent with the known profile of Cabometyx. Serious side effects were relatively common, and included high blood pressure, fatigue, diarrhea, and blood clots. Most patients given the therapy required dose modifications to manage its side effects.
It was encouraging to see that [Cabometyx] resulted in significant delays in disease progression in the CABINET trial — regardless of primary tumor site and grade.
The CABINET study was sponsored by the National Cancer Institute, part of the National Institutes of Health. It was led by scientists at the Alliance for Clinical Trials in Oncology, a network of cancer researchers dedicated to finding new and better treatments.
Peterson offered Exelixis’ thanks to all who played a role in the treatment’s advancement for pancreatic cancer.
“I would like to extend our sincere gratitude to the Alliance for Clinical Trials in Oncology for conducting the CABINET trial, to the FDA for [its] collaboration on the review of this application and to all the patients and physicians who participated in this important study,” Peterson said.
The FDA’s approval of Cabometyx for pancreatic and nonpancreatic NET was welcomed by The Healing NET Foundation, an advocacy group focused on this class of cancers.
“As a person who has lived with neuroendocrine tumors for over 14 years — and who has met many patients and caregivers in that time — I know that there are many challenges that come with this diagnosis, including the need to closely monitor the disease and adapt your treatment approach if faced with progression,” said Cindy Lovelace, the foundation’s cofounder. “As very few targeted therapies have been approved for advanced NET in recent years, I am excited that Cabometyx brings new hope to the patients in our community who have been in need of effective new treatment options.”