Last reviewed · How we verify

Treatment with vismodegib

University Hospital, Bordeaux · FDA-approved active Small molecule

Vismodegib inhibits the Hedgehog signaling pathway by blocking Smoothened, a key transmembrane protein required for pathway activation.

Vismodegib inhibits the Hedgehog signaling pathway by blocking Smoothened, a key transmembrane protein required for pathway activation. Used for Locally advanced basal cell carcinoma, Metastatic basal cell carcinoma.

At a glance

Generic nameTreatment with vismodegib
SponsorUniversity Hospital, Bordeaux
Drug classHedgehog pathway inhibitor
TargetSmoothened (SMO)
ModalitySmall molecule
Therapeutic areaOncology
PhaseFDA-approved

Mechanism of action

Vismodegib is a small-molecule antagonist of Smoothened (SMO), a G-protein coupled receptor essential for Hedgehog pathway signaling. By blocking SMO, it prevents activation of downstream transcription factors (GLI1/GLI2) that drive proliferation in Hedgehog-dependent cancers. This mechanism is particularly effective in basal cell carcinoma, where aberrant Hedgehog signaling is a primary driver of tumorigenesis.

Approved indications

Common side effects

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape: