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Cabozantinib
Source: Childhood Multiple Endocrine Neoplasia (MEN) Syndromes Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 3, 2025 · Captured 2026-09-09.
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Cabozantinib is a tyrosine kinase inhibitor (TKI) that targets three relevant pathways in medullary thyroid carcinoma: MET, VEGFR2, and RET.
In a phase I study, cabozantinib demonstrated promising clinical activity in a cohort of heavily pretreated patients with medullary thyroid carcinoma.[23]
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A double-blind phase III trial compared cabozantinib with placebo in adults with progressive, metastatic medullary thyroid carcinoma.[24]
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The estimated PFS was 11.2 months for patients who received cabozantinib and 4 months for patients who received a placebo.
At 1 year, 47.3% of patients who were treated with cabozantinib were alive and progression free, compared with 7.2% of patients who received a placebo.
Significant adverse effects resulted in dose reductions in 79% of patients and discontinuation of cabozantinib in 16% of patients.
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