Vismodegib

DB08828ApprovedInvestigationalSmall moleculeHedgehog Pathway Inhibitor

Vismodegib is a hedgehog pathway inhibitor used to treat patients with locally advanced or metastatic basal cell carcinoma. Vismodegib is an orally active small molecule that inhibits the hedgehog signaling pathway by binding to and inhibiting the transmembrane protein smoothened homologue (SMO).

Mechanism: During embryogenesis, the Hedgehog signaling pathway plays an important role in cell growth, differentiation apoptosis and self-renewal. After this developmental stage, the Hedgehog signaling pathway is silenced in all cells and tissues, except for hair, skin and stem cells. Mutations on elements of the Hedgehog signaling pathway may result in uncontrolled proliferation of basal skin cells, and dysregulated or aberrant Hedgehog signaling has been associated with the pathogenesis of basal cell carcinoma. Therefore, drugs that target and block this pathway, such as vismodegib, may be used to treat this condition. Vismodegib binds to and inhibits the transmembrane protein smoothened homologue (SMO), a protein that leads to the activation and nuclear translocation of several factors involved in the Hedgehog signaling pathway, inhibiting the activation of downstream Hedgehog target genes.
Curator reviewed · 2 references
Primary indication: Vismodegib is indicated for the treatment of adults with metastatic basal cell carcinoma, or with locally advanced basal cell carcinoma that has recurred following surgery or who are not candidates for surgery and who are...Curator reviewed · 3 structured indications
Formula / weight: C₁₉H₁₄Cl₂N₂O₃S · 421.297 g/mol (avg)
First approval: Canada, 2013 · United States, 2012 · European Union, 2020
Code names: GDC-0449 · NSC-747691 · NSC-755986 · RG-3616
Brand names: Erivedge