ABSTRACT
Introduction
Hepatocellular carcinoma (HCC) is one of the most frequent tumors affecting the gastrointestinal tract and a universal cause of morbidity and mortality. Cabozantinib is a strong multi-inhibitor of receptor tyrosine kinases approved for renal cell carcinoma that could be useful also for the treatment of HCC.
Areas covered
This review describes the chemical structure, the pharmacologic properties and current knowledge of the efficacy of cabozantinib in the treatment of HCC based on data available from first phase and later phase clinical trials. The ongoing studies testing cabozantinib, either alone or in combination with other drugs, are also described.
Expert opinion
Despite the recent achievements in the use of cabozantinib for patients diagnosed with hepatocellular carcinoma, data are still needed to allow clinicians to make better decisions on how to treat specific patient subgroups.
Article highlights
Cabozantinib is an oral agent that inhibits several receptor tyrosine kinases, including MET, VEGFR 1–3, AXL and RET
Cabozantinib has statistically significantly prolonged both overall and progression-free survival in advanced HCC patients previously treated with sorafenib
In the phase III CELESTIAL trial, cabozantinib was superior to placebo in all patient subgroups and efficacy endpoints
Cabozantinib is a new second-line treatment option and, for advanced HCC patients, the only third-line treatment option
Cabozantinib is normally well tolerated with manageable adverse events
Declaration of interest
The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Reviewer disclosures
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.