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Updated: May 19, 2026

Cell Population Analyses During Skin Carcinogenesis
Published on: August 21, 2013
Vismodegib: a promising drug in the treatment of basal cell carcinomas
1Sint-Augustinus Hospital, Oosterveldlaan 24, Antwerp, Belgium. luc.dirix@gza.be
Abstract:
Hedgehog pathway signaling is important for embryonic development; however, inappropriate reactivation of this pathway in adults has been linked to several forms of cancer. Vismodegib (Erivedge™), a first-in-class hedgehog pathway inhibitor, blocks the pathway by inhibiting the activity of the signaling protein SMO. Preclinical studies have provided promising indications of potential tumor-reducing activity in several cancers. Thus far, clinical pharmacology and Phase I studies have demonstrated the unique pharmacokinetic profile of vismodegib, its efficacy in certain types of tumors and a generally tolerable adverse-event profile. A pivotal Phase II clinical trial confirmed the favorable benefit:risk profile of vismodegib in advanced basal cell carcinoma.
Insights
Vismodegib is a novel hedgehog pathway inhibitor demonstrating efficacy in treating advanced basal cell carcinoma. Clinical trials confirm its favorable safety and risk profile for cancer patients.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Hedgehog pathway signaling is crucial for embryonic development.
- Aberrant reactivation of this pathway is implicated in various adult cancers.
- Vismodegib targets the SMO protein to inhibit hedgehog pathway activity.
Purpose of the Study:
- To evaluate the clinical pharmacology and efficacy of vismodegib.
- To assess the safety and tolerability profile of vismodegib.
- To confirm the benefit:risk profile of vismodegib in advanced basal cell carcinoma.
Main Methods:
- Preclinical studies indicated potential anti-tumor activity.
- Clinical pharmacology and Phase I studies assessed drug properties and initial efficacy.
- A pivotal Phase II clinical trial evaluated vismodegib in advanced basal cell carcinoma patients.
Main Results:
- Vismodegib exhibits a unique pharmacokinetic profile.
- The drug demonstrated efficacy in specific tumor types.
- Vismodegib generally presented a tolerable adverse-event profile.
- Phase II trial confirmed a favorable benefit:risk ratio for advanced basal cell carcinoma.
Conclusions:
- Vismodegib is an effective hedgehog pathway inhibitor for advanced basal cell carcinoma.
- The drug offers a favorable benefit:risk profile, supporting its clinical use.
- Vismodegib represents a significant advancement in targeted cancer therapy.
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