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Review
. 2020 Jun:86:101993.
doi: 10.1016/j.ctrv.2020.101993. Epub 2020 Feb 20.

Evolving role of regorafenib for the treatment of advanced cancers

Axel Grothey  1 , Jean-Yves Blay  2 , Nick Pavlakis  3 , Takayuki Yoshino  4 , Jordi Bruix  5
Affiliations
Free article
Review

Evolving role of regorafenib for the treatment of advanced cancers

Axel Grothey et al. Cancer Treat Rev. 2020 Jun.
Free article

Abstract

Regorafenib is an oral tyrosine kinase inhibitor (TKI) approved for the treatment of refractory metastatic colorectal cancer (mCRC), advanced gastrointestinal stromal tumors (GIST) previously treated with imatinib and sunitinib, and unresectable hepatocellular carcinoma (HCC) following progression on sorafenib. Regorafenib was initially approved for mCRC based on improved overall survival (OS) in the randomized, placebo-controlled, phase 3 CORRECT trial, which was confirmed in an expanded population of Asian patients in the randomized, placebo-controlled phase 3 CONCUR trial. Approvals in GIST, and more recently in HCC, were based on the results from the randomized, placebo-controlled, phase 3 GRID and RESORCE trials, respectively. In this review, we provide a comprehensive summary of the clinical evidence for approval of regorafenib in mCRC, GIST, and HCC, present emerging evidence of regorafenib activity in other tumor types (namely, gastroesophageal cancer, sarcomas, biliary tract cancer, and glioblastoma), and discuss trials in progress within the context of regorafenib's mechanism of action. We describe recent advances and key lessons learned with regorafenib, including the importance of managing common drug-related toxicities using dose-optimization strategies, the search for biomarkers to predict response to treatment, and highlight some of the unaddressed questions and future directions for regorafenib across tumors.

Keywords: Efficacy; Gastrointestinal cancer; Glioblastoma; Regorafenib; Safety; Sarcoma.

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Conflict of interest statement

Declaration of Competing Interest J-YB has received grants and personal fees from Bayer, GlaxoSmithKline, Novartis, and Roche; JB has received consulting fees from AbbVie, Adaptimmune, ArQule, Astra-Medimmune, Basilea, Bayer, Bio-Alliance, Bristol-Myers Squibb, BTG, Eisai, Gilead, Incyte, Ipsen, Kowa, Lilly, Merck Sharp and Dohme, Nerviano, Novartis, Quirem, Roche, Sanofi, Sirtex, and Terumo, grants from Bayer and BTG, paid conference attendance from Bayer, BTG, and Ipsen, and presentation fees from Bayer, BTG, Eisai, Ipsen, Sirtex, and Terumo; AG has received grants, honoraria, and non-financial support from Array BioPharma, Bayer, Daiichi Sankyo, and Genentech/Roche; NP has received personal fees from Amgen, AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Ipsen, Merck KgA, Merck Serono, Merck Sharp and Dohme, Novartis, Pfizer, Roche, and Takeda, grants from Bayer and Pfizer, and has other relationships to disclose with Roche; TY has received grants from Chugai Pharma, Daiichi Sankyo, GlaxoSmithKline, Merck Sharp and Dohme, Novartis, Ono Pharmaceutical, Parexel, Sanofi, and Sumitomo Dainippon Pharma.

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