Colorectal cancer (CRC) is the third most common malignant neoplasm among men and the second most common among women. According to the World Cancer Report, the number of people suffering from this disease is growing steadily. In 2012, there were more than 1.36 million new cases of CRC, and approximately 694,000 people died from this disease worldwide.

A sensitive literature search identified 12 relevant publications, including: a CORRECT phase III study assessing the effect of regorafenib in patients with metastatic CRC that continued to progress despite using all standard treatment methods; a CONCUR Phase III study evaluating the clinical effect of regorafenib in Asian patients with metastatic CRC; a CONSIGN study conducted after the CORRECT and CONCUR studies that assessed the safety profile of regorafenib prior to market entry; and various systematic reviews evaluating the safety of regorafenib.

The efficacy and safety of regorafenib for treating patients with metastatic CRC was evaluated in two major clinical studies: CORRECT and CONCUR. Although the studies were randomized, double-blind, and placebo-controlled, they were conducted in different patient populations. Before treatment with regorafenib, patients received, depending on the country, fluoropyrimidines, oxaliplatin, irinotecan, or bevacizumab, and patients with the wild-type KRAS gene also received cetuximab and panitumumab. Results from both studies indicated that regorafenib had a clinically significant positive effect on rates of progression-free survival and overall survival in patients with treatment-resistant metastatic CRC.

Regorafenib can be recommended as a monotherapy for resistant metastatic CRC when there are no contraindications to use. Considering the safety profile of regorafenib, further research is needed to determine the best dosage of regorafenib and the most appropriate clinical and molecular biomarkers for determining which patients would benefit most from this treatment.