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Exelixis, Inc. EXEL announced that the FDA has approved the label expansion of its lead drug Cabometyx (cabozantinib).
The regulatory body approved Cabometyx for the treatment of adult and pediatric patients 12 years of age and older with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic neuroendocrine tumors (pNET).
Simultaneously, the drug was approved for adult and pediatric patients (12 years of age and older) with previously treated, unresectable, locally advanced or metastatic, well-differentiated extra-pancreatic NET (epNET).
Consequently, Cabometyx is now the first and only systemic treatment that is FDA-approved for previously treated neuroendocrine tumors regardless of primary tumor site, grade, somatostatin receptor expression and functional status.
The approval came ahead of the target action date of April 3, 2025.
More on EXEL’s Cabometyx
Cabometyx is one of the leading tyrosine kinase inhibitor (TKI) for the treatment of renal cell carcinoma (RCC) in both the frontline immuno-oncology (IO) +TKI market and the second-line monotherapy segment.
We note that Cabometyx has also been approved for use in combination with Bristol Myers’ BMY Opdivo in the first-line setting in RCC. BMY’s Opdivo is one of the leading IO drugs, and it has been approved for various oncology indications.
Cabometyx is also approved for the treatment of hepatocellular carcinoma.
The drug is approved for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible.
The latest FDA approval is based on results from the phase III CABINET study, which evaluated Cabometyx compared with placebo in two cohorts of patients with previously treated NET: advanced pNET and advanced epNET.
Results demonstrated a statistically significant and clinically meaningful improvement in progression-free survival compared to placebo.
NET are mostly heterogeneous tumors that originate from the neuroendocrine cells of the digestive tract and other organs, such as the lung and pancreas. The patients diagnosed with the disease at an advanced stage face a poor prognosis.
The label expansion of Cabometyx is expected to boost sales of the drug, which reached $1.8 billion in 2024.
EXEL Makes Encouraging Pipeline Progress
The pipeline progress has been impressive as well, as Exelixis looks to expand its oncology portfolio beyond Cabometyx.
The company is now focused on developing zanzalintinib, a next-generation oral TKI. In January 2025, results from an expansion cohort of the phase Ib/II STELLAR-001 study evaluating zanzalintinib alone or in combination with Tecentriq (atezolizumab) in patients with previously treated metastatic colorectal cancer (CRC) were presented. The data support zanzalintinib’s ongoing pivotal development in metastatic CRC.