Vismodegib: A Hedgehog Pathway Inhibitor for Locally Advanced and Metastatic Basal Cell Carcinomas

Basal cell carcinomas (BCCs) are the most common cancer in the United States, and the overwhelming majority of BCCs are the result of hedgehog pathway activation. While locally advanced and metastatic BCC are rare, currently available treatments remain limited and are often unsuccessful. Vismodegib inhibits a key regulatory protein in the hedgehog pathway and was approved by the United States Food and Drug Administration in 2012. This orally-administered medication offers a novel approach for treating locally advanced and metastatic BCC. The following review will address vismodegib's mechanism of action, published clinical trial data, and the questions that still remain unanswered about this new medication.