Medullary Thyroid Carcinoma Medication

Updated: Jun 14, 2022
  • Author: Anastasios K Konstantakos, MD; Chief Editor: Neerav Goyal, MD, MPH, FACS  more...
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Medication Summary

Although surgery remains the standard treatment for medullary thyroid carcinoma (MTC), several tyrosine kinase inhibitors (TKIs) are approved by the US Food and Drug Administration (FDA) for treatment of advanced or metastatic MTC. These include the multitarget TKIs vandetanib and cabozantinib, and the RET tyrosine kinase inhibitors selpercatinib and pralsetinib. (See Treatment)


Tyrosine Kinase Inhibitors

Class Summary

These agents target various tyrosine kinases including MET, RET, and VEGFR-2.

Vandetanib (Caprelsa)

Tyrosine kinase inhibitor (TKI) with selective activity against RET, VEGFR-2, and EGFR. Indicated for treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease.

Cabozantinib (Cometriq)

Cabozantinib is a tyrosine kinase inhibitor that targets RET, MET, VEGFR-1, -2, and -3, KIT, TrkB, FLT-3, AXL, and TIE-2 pathways; these tyrosine kinases are involved in both normal cellular function and pathologic processes (eg, oncogenesis, metastasis, tumor angiogenesis, and maintenance of tumor microenvironment). It is indicated for treatment of progressive, metastatic medullary thyroid cancer.


RET Kinase Inhibitors

Class Summary

Genomic alterations in rearranged during transfection (RET) kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth.

Selpercatinib (Retevmo)

Selpercatinib is a kinase inhibitor of wild-type rearranged during transfection (RET) and multiple mutated RET isoforms, as well as vascular endothelial growth factor receptors (VEGFR1, VEGFR3). It is indicated for advanced or metastatic RET-mutant MTC in adults and children aged 12 years or older who required systemic therapy.

Pralsetinib (Gavreto)

A once-daily therapy designed to selectively target RET alterations. Indicated for advanced or metastatic rearranged during transfection (RET)-mutant MTC in adult and pediatric patients ≥12 years who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).