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Expert Opinion on Orphan Drugs Volume 3, 2015 - Issue 12

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Drug Evaluations

Cabozantinib: an orphan drug for thyroid cancer

D. ViolaEndocrine Unit, Department of Clinical and Experimental Medicine, University of Pisa, 8th Building, Ospedale Cisanello, -Via Paradisa, 2, 56124Pisa, Italy

V. CappagliEndocrine Unit, Department of Clinical and Experimental Medicine, University of Pisa, 8th Building, Ospedale Cisanello, -Via Paradisa, 2, 56124Pisa, Italy

A. MatroneEndocrine Unit, Department of Clinical and Experimental Medicine, University of Pisa, 8th Building, Ospedale Cisanello, -Via Paradisa, 2, 56124Pisa, Italy

S. MazzeoRadiology Unit, Department of Translational Research on New Technologies in Medicine and Surgery, University of Pisa, 56124Pisa, Italy

R. EliseiEndocrine Unit, Department of Clinical and Experimental Medicine, University of Pisa, 8th Building, Ospedale Cisanello, -Via Paradisa, 2, 56124Pisa, ItalyCorrespondence[email protected]

Pages 1469-1477 | Published online: 20 Nov 2015

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https://doi.org/10.1517/21678707.2015.1112789
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*The first study that investigated the use and the effectiveness of cabozantinib in MTC patients

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**The study that confirmed the efficacy in terms of progression-free survival (PFS) prolongation, safety and tolerability of cabozantinib in a large cohort of progressive MTC patients and led to its approval by FDA and EMA

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**The study that demonstrated the prolongation of the overall survival (OS) in a subset of patients with a tumor that harbor RET M918T mutation

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Cabozantinib: an orphan drug for thyroid cancer

Bibliography

*The first study that investigated the use and the effectiveness of cabozantinib in MTC patients

**The study that confirmed the efficacy in terms of progression-free survival (PFS) prolongation, safety and tolerability of cabozantinib in a large cohort of progressive MTC patients and led to its approval by FDA and EMA

**The study that demonstrated the prolongation of the overall survival (OS) in a subset of patients with a tumor that harbor RET M918T mutation

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Cabozantinib: an orphan drug for thyroid cancer

Bibliography

*The first study that investigated the use and the effectiveness of cabozantinib in MTC patients

**The study that confirmed the efficacy in terms of progression-free survival (PFS) prolongation, safety and tolerability of cabozantinib in a large cohort of progressive MTC patients and led to its approval by FDA and EMA

**The study that demonstrated the prolongation of the overall survival (OS) in a subset of patients with a tumor that harbor RET M918T mutation

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