New data demonstrate the anti-tumor activity of vismodegib (Erivedge, Genentech) 150mg daily in metastatic and locally advanced basal cell carcinoma (BCC). (N Engl J Med; 366:2171-2179) The study found that the independently assessed response rate was 30 percent in patients with metastatic BCC (n=33) and 43 percent in patients with locally advanced BCC (n=63). Twenty-one percent of all patients had complete response. The median duration of response was 7.6 months in both cohorts. Vismodegib is a first-in-class, small-molecule inhibitor of the hedgehog pathway. The multicenter, international, two-cohort, nonrandomized study enrolled patients with metastatic BCC and those with locally advanced BCC who had inoperable disease or for whom surgery was inappropriate (because of multiple recurrences and a low likelihood of surgical cure, or substantial anticipated disfigurement). Adverse events occurring in more than 30 percent of patients were muscle spasms, alopecia, dysgeusia (taste disturbance), weight loss, and fatigue. Serious adverse events were reported in 25 percent of patients; seven deaths due to adverse events were noted.