Summary

Vismodegib is the first oral hedgehog pathway inhibitor approved by the European Medicines Agency for the treatment of adults with symptomatic metastatic basal cell carcinoma (mBCC) or locally advanced basal cell carcinoma (laBCC), both of which are not suitable for treatment with surgery or radiotherapy. The pivotal Erivance BCC trial [NCT00833417] was an international 2-cohort nonrandomized study of oral vismodegib (150 mg daily) in patients with laBCC or mBCC. This article presents the safety and investigator-assessed efficacy results of the analysis, performed 30 months after the primary analysis (May 30, 2013, data cutoff).