The safety and efficacy of sonidegib for the treatment of locally advanced basal cell carcinoma

Expert Rev Anticancer Ther. 2016 Oct;16(10):1011-8. doi: 10.1080/14737140.2016.1230020. Epub 2016 Sep 22.


Introduction: Basal cell carcinomas (BCCs) are the commonest malignancy in the Western world. Locally advanced BCCs (laBCCs) represent tumours that have developed in difficult-to-treat facial sites, aggressively recurrent tumours, large neglected tumours and those in which current treatment options are excluded by clinical or patient-driven criteria. It is estimated laBCCs represent 1% of BCCs.

Areas covered: Sonidegib is an oral hedgehog pathway inhibitor with a novel structure. It has recently been licensed for the treatment of laBCC. This article provides a comprehensive review of the literature regarding sonidegib, detailing the pharmacology of the compound, clinical trial data, competitor compounds and a future perspective. Expert commentary: Sonidegib is a novel smoothened (SMO) inhibitor with comparable efficacy to vismodegib, with patient response rates of 44% (sonidegib) and 43% (vismodegib). The adverse effect profile of these two treatments is similar with the main effects being considered to be class effects of SMO inhibitors.

Keywords: Basal cell carcinoma; hedgehog pathway inhibitors; locally advanced basal cell carcinoma; small-molecule inhibitors; smoothened inhibitors; sonidegib.

Publication types

  • Comparative Study
  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Biphenyl Compounds / adverse effects
  • Biphenyl Compounds / pharmacology
  • Biphenyl Compounds / therapeutic use*
  • Carcinoma, Basal Cell / drug therapy*
  • Carcinoma, Basal Cell / pathology
  • Hedgehog Proteins / metabolism
  • Humans
  • Neoplasm Recurrence, Local
  • Pyridines / adverse effects
  • Pyridines / pharmacology
  • Pyridines / therapeutic use*
  • Skin Neoplasms / drug therapy*
  • Skin Neoplasms / pathology
  • Smoothened Receptor / antagonists & inhibitors


  • Antineoplastic Agents
  • Biphenyl Compounds
  • Hedgehog Proteins
  • Pyridines
  • SMO protein, human
  • Smoothened Receptor
  • sonidegib