FDA Accepts Supplemental New Drug Application for Cabozantinib in GEP-NETs

The US Food and Drug Administration (FDA) has accepted the supplemental new drug application from Exelixis, Inc. for cabozantinib to treat patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs).

Cabozantinib can be used for the treatment of patients with previously treated, locally advanced, unresectable or metastatic, well- or moderately differentiated pancreatic neuroendocrine tumors (pNETs) or those with previously treated, locally advanced, unresectable or metastatic, well- or moderately differentiated extra-pancreatic neuroendocrine tumors (epNETs).

The treatment was also granted orphan drug designation to treat patients with pNETs, as well as a standard review with a Prescription Drug User Fee Act target action date of April 3, 2025.

The FDA based their decision on results from the phase 3 CABINET trial, which investigated the potential benefits of cabozantinib compared with placebo in patients with previously treated NETs, advanced pNETs, and advanced epNETs. The study was unblind, and patients in the placebo cohort were provided the option to switch to the cabozantinib cohort.

CABINET ended early due to a significant improvement in progression-free survival (PFS) upon interim analysis in both treatment arms.

Researchers concluded that treatment with cabozantinib resulted in statistically significant and clinically meaningful improvement in PFS with cabozantinib compared with placebo.

“The FDA’s acceptance of this application marks another important milestone in our commitment to bringing cabozantinib to patients living with difficult-to-treat cancers and who have limited treatment options,” said Amy Peterson, MD, executive vice president, product development and medical affairs, and chief medical officer at Exelixis. “We appreciate the opportunity to work with the FDA in the coming months as they review our application, with the goal to bring this new, effective treatment option to patients with advanced neuroendocrine tumors as quickly as possible.”