title: Efficacy and safety of vismodegib : a new therapeutic agent in the treatment of basal cell carcinoma.
abstract: BACKGROUND
  Basal cell carcinoma (BCC) is the most common human malignancy. Treatment options for the minority of patients presenting with locally advanced inoperable or metastatic BCC are very limited. The hedgehog (Hh) pathway plays a crucial role in the pathogenesis of BCC. Recent advances in targeting this pathway have led to the development of a first-in-class, small-molecule oral Hh inhibitor, vismodegib (Erivedge®, Genentech).
  
  
  METHODS
  In this article, we review vismodegib with regard to its mechanism of action, clinical efficacy, safety and tolerability, and we consider the causes of emerging resistance to the drug.
  
  
  CONCLUSIONS
  Vismodegib is a welcome addition to the treatment paradigm for BCC. Approval was based on Phase II evidence, the patient number was relatively small, there was no control group or a comparator group and survival data have not been presented so longer term follow-up and larger exposure to the drug is required to fully appreciate its clinical utility into the future. With ongoing use of the drug in the nontrial population and further studies investigating its use in both early- and later-stage disease, we will get a better understanding of the drug and determine its place in the armamentarium against BCC.
authors.affiliation: Mater Misericordiae University Hospital, University College Dublin, Department of Medical Oncology , Eccles Street, Dublin 7 , Ireland.
authors.forename: Tomas G
authors.initials: TG
authors.lastname: Lyons
authors.forename: Grainne M
authors.initials: GM
authors.lastname: O'Kane
authors.forename: Catherine M
authors.initials: CM
authors.lastname: Kelly
mesh_terms: D000813:Anilides / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use; D002280:Basal Cell Carcinoma / Q000188:drug therapy* / Q000473:pathology; D017277:Drug Approval; D019008:Drug Resistance, Neoplasm; D053823:Hedgehog Proteins / Q000037:antagonists & inhibitors; D006801:Humans; D009362:Neoplasm Metastasis; D011725:Pyridines / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000473:pathology
chemical_list: D000813:Anilides; D000970:Antineoplastic Agents; D053823:Hedgehog Proteins; C538724:HhAntag691; D011725:Pyridines
keywords: basal cell carcinoma; hedgehog pathway; vismodegib