Definition

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.