Exelixis has received the US Food and Drug Administration (FDA) expanded approval for Cabometyx (cabozantinib) to include advanced neuroendocrine tumours (NET).
Cabometyx tablets are already approved in the US for several conditions, including advanced renal cell carcinoma, hepatocellular carcinoma, and differentiated thyroid cancer.
The expanded approval covers both pancreatic and extra-pancreatic NET (epNET) that are unresectable, locally advanced, or metastatic, in patients aged 12 and older.
NET are tumours originating from neuroendocrine cells in organs like the pancreas and lungs, often resulting in a poor prognosis.
The medication is also authorised in more than 65 countries outside the US and Japan.
Exelixis chief medical officer and product development and medical affairs executive vice president Amy Peterson said: “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 tumours.
“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 their collaboration on the review of this application and to all the patients and physicians who participated in this important study.
“Looking forward, we are doubling down on our commitment to the NET community as we prepare to initiate our STELLAR-311 pivotal trial examining zanzalintinib versus everolimus in the first half of 2025.”
The FDA’s decision is based on the results of the CABINET Phase 3 trial, which compared Cabometyx with a placebo in patients with advanced pancreatic and extra-pancreatic NET.
In January 2025, the National Comprehensive Cancer Network updated its guidelines to include Cabometyx as a preferred regimen for well-differentiated advanced NET.
The safety profile of Cabometyx in the CABINET trial matched its known safety characteristics, with no new safety signals detected.
However, hypertension was more prevalent in NET patients compared to other tumour types. Most patients required dose adjustments to manage side effects.
In 2016, Exelixis granted French pharmaceutical firm Ipsen rights for the commercialisation and further development of Cabometyx outside the US and Japan.
In 2017, the company granted Japanese drugmaker Takeda exclusive rights for the development and commercialisation of Cabometyx in Japan, retaining US rights.
CABINET trial study chair Jennifer Chan said: “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.
“It was encouraging to see that cabozantinib resulted in significant delays in disease progression in the CABINET trial, regardless of primary tumour site and grade.
“This FDA approval marks a meaningful advancement, which may establish an important new treatment option for patients, without limitations based on somatostatin receptor expression and functional status.”
