Home > D. Systemic pathology > Toxics and drugs > Therapeutics > Targeted therapy > Small molecules > vismodegib


Thursday 20 December 2012



Vismodegib acts as a cyclopamine-competitive antagonist of the smoothened receptor (SMO) which is part of the hedgehog signaling pathway (SHH pathway). Cyclopamine is a naturally occurring SMO antagonist.

SMO inhibition causes the transcription factors GLI1 and GLI2 to remain inactive, which prevents the expression of tumor mediating genes within the hedgehog pathway.

This pathway is pathogenetically relevant in more than 90% of basal-cell carcinomas.

Vismodegib (trade name Erivedge) is a drug for the treatment of basal-cell carcinoma (BCC).

The approval of vismodegib on January 30, 2012, represents the first Hedgehog signaling pathway targeting agent to gain U.S. Food and Drug Administration (FDA) approval.

The drug is also undergoing clinical trials for metastatic colorectal cancer, small-cell lung cancer, advanced stomach cancer, pancreatic cancer, medulloblastoma and chondrosarcoma as of June 2011.

The drug was developed by the biotechnology / pharmaceutical company Genentech, which is headquartered at South San Francisco, California, USA.


Vismodegib is indicated for patients with basal cell carcinoma (BCC), which has metastasized to other parts of the body, relapsed after surgery, or cannot be treated with surgery or radiation.