A substance being studied in the treatment of cancer. It blocks a type of protein involved in tissue growth and repair and may block the growth of cancer cells. It is a type of Hedgehog signaling pathway antagonist.

An orally bioavailable, small molecule inhibitor of SMO and the Hedgehog (Hh) pathway, with potential antineoplastic activity. Upon oral administration, vismodegib targets, binds to and inhibits the cell membrane-spanning G-protein coupled receptor SMO, which may result in the suppression of Hh pathway signaling and a decrease in tumor cell proliferation and survival. SMO is activated upon binding of Hh ligand to the cell surface receptor Patched (PTCH); inappropriate activation of Hh signaling and uncontrolled cellular proliferation may be associated with SMO mutations.