Targeted therapies by means of compounds that inhibit a specific target molecule represent a new perspective in the treatment of cancer. Various signaling pathways have been implicated in the development and progression of cancer. There is a general agreement that molecules interfering simultaneously with multiple targets might be more effective than single target agents. Regorafenib ( BAY 73-4506) is a novel, oral multikinase inhibitor, which demonstrates a broad spectrum of antitumor activity, probably due to the targeting of several angiogenic, oncogenic and stromal kinases. On September 2012, regorafenib was approved by US Food and Drug Administration( FDA) for previously heavy treated metastatic colorectal cancer. On February 2013, US FDA expanded the approved use to treat patients with advanced gastrointestinal stromal tumors. Two large, randomized,international multicentre,phase Ⅲ clinical trial in patients with metastatic colorectal cancer and gastrointestinal stromal tumour treated by regorafenib were finished. The CORRECT tri-al evaluated regorafenib for the treatment of patients with metastatic colorectal cancer who had progressed after standard therapies. The results confirmed that regorafenib was associated with significant improvements in overall survival. The GRID trial showed that rego-rafenib could provide a significant improvement in progression-free survival compared with placebo in patients with metastatic gastroin- testinal stromal tumour following failure of imatinib and sunitinib. The toxicity profile of regorafenib is comparable with other oral mul-tikinase inhibitors with similar molecular targets. Most toxicities associated with regorafenib are mild/moderate and clinically managable. Further extensive clinical development as a single agent or in combination with standard chemotherapeutic agents in various malignant tumors is ongoing.%分子靶向治疗是近年来肿瘤治疗领域的研究热点。由于大多数肿瘤的生物学行为由多个信号传导通路共同发挥作用，故普遍认为针对多靶点进行分子靶向治疗有可能获得更好的疗效。瑞戈非尼（ Regorafenib，BAY 73-4506）是一种新型的口服多靶点磷酸激酶抑制剂，能阻断肿瘤细胞增殖、抑制肿瘤血管生成、调控肿瘤微环境，具有良好的抗肿瘤活性。2012年9月，瑞戈非尼获得美国食品药品监督管理局（ FDA）批准，用于治疗既往接受过标准治疗的转移性结直肠癌。2013年2月，瑞戈非尼获批准用于进展期胃肠间质瘤。目前已完成了两项瑞戈非尼大型随机国际性多中心Ⅲ期临床试验，分别是针对转移性结直肠癌和胃肠间质瘤。 CORRECT研究首次证实，小分子激酶抑制剂瑞戈非尼在标准治疗失败后的转移性结直肠癌中能达到总生存获益。 GRID研究提示，对于伊马替尼和舒尼替尼治疗后疾病进展的转移性或不可切除胃肠间质瘤患者，瑞戈非尼较安慰剂可显著改善无进展生存期，为这一难治性患者群带来获益。瑞戈非尼的不良反应与其他类似的口服多靶点激酶抑制剂相似，大多数不良反应为轻、中度，可以通过临床处理得到控制。瑞戈非尼单药或与标准化疗药物联合治疗其他恶性肿瘤的临床研究目前正在积极开展中。
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