What is the role of vismodegib in the management of basal cell carcinoma (BCC)?

Updated: Mar 02, 2020
  • Author: Robert S Bader, MD; Chief Editor: William D James, MD  more...
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Vismodegib (Erivedge) is the first FDA-approved drug for advanced forms of basal cell carcinoma (BCC). It selectively inhibits Smoothened (SMO), a key transmembrane protein involved in hedgehog signal transduction of cancerous epithelial cells. In a phase I dose-ranging study by Von Hoff et al, 18 of 33 patients showed an objective response. Two of the 18 had complete response, and the remaining 16 showed a partial response. [97] FDA approval was based on a single, international, open-label trial (n=104). Of the 104 participants, 96 were evaluable. Of those with metastatic BCC (n=33), 30.3% had partial response, but none had complete response. With locally advanced BCC (n=63), 22% showed a partial response and 20% showed complete response. [98]

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