IMPORTANCE OF THE FIELD: The successful approval of sorafenib has greatly stimulated the development of other molecular targeted agents in advanced hepatocellular carcinoma (HCC).
AREAS COVERED IN THIS REVIEW: The aim of this review was to summarize the key data of sorafenib Phase III studies and the lessons and unanswered questions with sorafenib in HCC. It extensively updated the current ongoing studies combining sorafenib with erlotinib and other targeted agents or chemotherapy, and many molecularly targeted agents under development that inhibit angiogenesis, EGFR, and mammalian target of rapamycin (mTOR).
WHAT THE READER WILL GAIN: A comprehensive understanding of the current status of drug development of targeted agents in advanced HCC and insights into the challenges of developing these agents in HCC.
TAKE HOME MESSAGE: Sorafenib is the first and only approved agent in advanced HCC. Several Phase III studies are ongoing with other drugs in advanced HCC. Many early clinical trials are conducted to assess other molecularly targeted agents that inhibit different pathways of hepatocarcinogenesis.