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Onco Targets Ther. 2016 Sep 14;9:5671-5678. eCollection 2016.

Sonidegib, a novel smoothened inhibitor for the treatment of advanced basal cell carcinoma.

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Department of Dermatology, University of Texas, McGovern Medical School.
Mohs Surgery Unit, Department of Dermatology; Department of Head and Neck Surgery, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.


Basal cell carcinoma (BCC) is the most common nonmelanoma skin cancer. If left untreated, BCCs can become locally aggressive or even metastasize. Currently available treatments include local destruction, surgery, and radiation. Systemic options for advanced disease are limited. The Hedgehog (Hh) pathway is aberrantly activated in a majority of BCCs and in other cancers. Hh pathway inhibitors are targeted agents that inhibit the aberrant activation of the Hh pathway, with smoothened being a targeted component. Sonidegib is a novel smoothened inhibitor that was recently approved by the US Food and Drug Administration. This review focuses on BCC pathogenesis and the clinical efficacy of sonidegib for the treatment of advanced BCC.


Hedgehog pathway; clinical trials; nonmelanoma skin cancer

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