Vandetanib

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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Context: Treatment of Childhood Multiple Endocrine Neoplasia (MEN) Syndromes / MEN Type 2 (MEN2) Syndromes / Targeted therapy

Vandetanib is a selective kinase inhibitor of RET, vascular endothelial growth factor receptor, and epidermal growth factor receptor.

A randomized phase III trial included adult patients with unresectable locally advanced or metastatic (hereditary or sporadic) medullary thyroid carcinoma who were treated with either vandetanib or placebo.[20]

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The study found that vandetanib administration was associated with significant improvements in progression-free survival (PFS), response rate, disease control rates, and biochemical response.

Children with locally advanced or metastatic medullary thyroid carcinoma were treated with vandetanib in a phase I/II trial.[21]

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Of 16 patients, only the one patient without the M918T RET variant had no response.

Of the 15 patients who had tumor responses, seven had partial responses.

Three of the 15 patients had subsequent disease recurrences.

Eleven of the 16 patients treated with vandetanib remained on therapy at the time of the report.

A subsequent follow-up analysis of this cohort plus one additional patient revealed that 10 of the 17 patients achieved partial responses, and an additional 6 individuals had stable disease. The median PFS for these patients was 6.7 years.[22]

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Preserved source evidence · Not medical advice