Drug intelligence / Profile preview

vismodegib

Development stage
Approved
Lead developer
Genentech
Modality
Small Molecules
Administration
Oral
01

Overview

Vismodegib is a small molecule inhibitor of the Hedgehog signaling pathway, specifically targeting the smoothened receptor (SMO). It is used primarily for the treatment of adults with advanced basal cell carcinoma (BCC), including cases that have metastasized, recurred after surgery, or are not amenable to surgery or radiation. Vismodegib works by binding to and antagonizing SMO, thereby blocking downstream activation of GLI transcription factors and inhibiting tumor growth driven by aberrant Hedgehog pathway activation. The drug was developed as the first FDA-approved agent targeting this pathway and is marketed under the brand name Erivedge.

Brand names
Erivedge
Other names
vismodegib
02

Targets

SMO (Smoothened)

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