**The US Food and Drug Administration (FDA) has approved Exelixis’ Cabometyx (cabozantinib) to treat advanced neuroendocrine tumours (NETs).**
The drug has been authorised for use in adult and paediatric patients aged 12 years and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic NETs or extra-pancreatic NETs.
With an estimated prevalence of 380,000 in the US last year, NETs are tumours that arise from the neuroendocrine cells of the digestive tract and other organs, including the lungs and pancreas.
Most NETs develop slowly over a number of years, but eventually all patients with advanced or metastatic NETs will experience refractory and progressive disease.
Cabometyx is designed to block the action of tyrosine kinase enzymes, which can be found in certain receptors in cancer cells and help to send growth signals. The drug is already approved in the US to treat certain cases of renal cell carcinoma, hepatocellular carcinoma and differentiated thyroid cancer.
The FDA’s latest decision was supported by positive results from the late-stage CABINET trial, which evaluated Cabometyx against placebo in patients with previously treated advanced pancreatic NETs or extra-pancreatic NETs.
Cabometyx demonstrated continued improvement in the primary endpoint of progression-free survival (PFS). Among the pancreatic NETs cohort, median PFS was 13.8 months for Cabometyx versus 4.4 months for placebo after a median follow-up of 13.8 months. For those with extra-pancreatic NETs, median PFS was 8.4 months and 3.9 months for Cabometyx and placebo, respectively, at a median follow-up of 10.2 months.
Study chair for the CABINET trial, Jennifer Chan, Dana-Farber Cancer Institute, said: “The characteristics of NETs vary widely from patient to patient, and very few treatment options have demonstrated the ability to improve outcomes across such a heterogeneous population.
“… 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.”
Exelixis holds the exclusive rights to develop and commercialise Cabometyx in the US, and has granted Ipsen exclusive rights to the drug outside of the US and Japan, and exclusive rights to Takeda in Japan.