FDA Approves Exelixis' Lead Cancer Drug Cabometyx For Expanded Use In Neuroendocrine Tumors

The U.S. Food and Drug Administration has approved Exelixis Inc.’s EXEL Cabometyx (cabozantinib) for two indications in adult and pediatric patients with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic and extra-pancreatic neuroendocrine tumors (NET).

NETs are heterogeneous tumors arising from the digestive tract’s neuroendocrine cells and other organs, such as the lung and pancreas.

The approval comes before the Prescription Drug User Fee Act action date of April 3.

The FDA approval — adding to five previous approvals for Cabometyx — is based on the CABINET Phase 3 trial results.

Final progression-free survival results were presented at the 2024 European Society for Medical Oncology Congress and published in The New England Journal of Medicine. 

In January 2025, the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology for Neuroendocrine and Adrenal Tumors were updated to include cabozantinib as a category 1 preferred regimen for the majority of well-differentiated advanced NET following specific treatments and as a category 2A preferred regimen for other forms of advanced NET, depending on tumor grade and different requirements for prior therapy.

In February, Exelixis announced the final results from the phase 3 CheckMate-9ER pivotal trial evaluating Cabometyx (cabozantinib) in combination with Bristol-Myers Squibb Co.’s BMY Opdivo (nivolumab) versus sunitinib for patients with previously untreated advanced renal cell carcinoma (RCC).

After more than five years of follow-up, the findings demonstrated that the efficacy benefits of Cabometyx in combination with Opdivo were sustained long-term.

At a median follow-up of 67.6 months, Cabometyx, in combination with Opdivo, improved progression-free survival and overall survival compared with Pfizer Inc’s PFE Sutent (sunitinib) in the intent-to-treat population.

EXEL Price Action: EXEL stock is down 2.31% at $36.83 at publication on Wednesday.

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