Vismodegib

Vismodegib is the first in a new class of orally active anti-cancer drugs that inhibit the hedgehog (Hh) signalling pathway by binding to the smoothened transmembrane protein and blocking induction of Hh target genes. Short-term studies have found that approximately 40% of patients with locally advanced BCC had a clinical response and that there was clearance of the tumour in 20% of patients. The median duration of response was 26.2 months. Tumour response should be gauged through serial measurements of tumour diameter and the documentation of the presence of ulceration. Therapy should be continued until disease progression or unacceptable side effect profile. The benefit gained and side effect profile associated with continued treatment should be regularly evaluated in each individual patient. For females of childbearing potential, pregnancy testing within 7 days before starting vismodegib and commencement of the manufacturer's pregnancy prevention programme are imperative.