# Vismodegib

Source: https://onco.cc/drugs/vismodegib/  
OnCo record `vismodegib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Vismodegib was the first hedgehog-pathway drug, for basal cell carcinomas too advanced for surgery; it shrinks most tumours but muscle cramps, taste loss and hair loss make long-term use hard.

## Summary

ERIVANCE (NEJM 2012): 30% response in metastatic and 43% in locally advanced BCC; long-term follow-up ~50-60% response in locally advanced disease. Teratogenic (pregnancy prevention programme). Also used in SHH medulloblastoma (adults) and studied neoadjuvantly (VISMONEO). Resistance via SMO mutations.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill
- Brand: Erivedge
- Modality: Small-molecule smoothened (hedgehog) inhibitor
- Mechanism: Binds and inhibits SMO, preventing GLI-mediated transcription downstream of PTCH1 loss.
- Approvals: US 2012: Metastatic BCC or locally advanced BCC recurrent after surgery or not amenable to surgery/radiation; EU 2013: Same

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Vismodegib
- ERIVANCE (NEJM 2012): https://doi.org/10.1056/NEJMoa1113713
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vismodegib

## Connected records

- cancers: [Basal cell carcinoma](https://onco.cc/cancers/basal-cell-carcinoma/), [Locally advanced and metastatic basal cell carcinoma](https://onco.cc/cancers/locally-advanced-bcc/), [Medulloblastoma](https://onco.cc/cancers/medulloblastoma/), [SHH-activated medulloblastoma](https://onco.cc/cancers/medulloblastoma-shh/), [Skin cancer (all types)](https://onco.cc/cancers/skin-cancer/)
- technologies: [Hedgehog pathway inhibitors](https://onco.cc/technologies/hedgehog-inhibitors/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [PTCH1 (Patched 1)](https://onco.cc/targets/ptch1/), [Smoothened (hedgehog pathway)](https://onco.cc/targets/smoothened/)
- companies: [Roche / Genentech](https://onco.cc/companies/roche-genentech/)
- terms: [Cancer stem cell theory and phenotypic plasticity](https://onco.cc/terms/cancer-stem-cell-theory/), [Medulloblastoma molecular groups (WNT, SHH, group 3, group 4)](https://onco.cc/terms/medulloblastoma-molecular-groups/), [Why people stop taking hedgehog inhibitors](https://onco.cc/terms/hedgehog-inhibitor-tolerability/)
- trials: [CUPISCO](https://onco.cc/trials/cupisco/), [DETERMINE](https://onco.cc/trials/determine-trial/), [ERIVANCE BCC](https://onco.cc/trials/erivance/), [MyPathway](https://onco.cc/trials/mypathway/), [SJMB12](https://onco.cc/trials/sjmb12/), [STEVIE (vismodegib in ordinary practice)](https://onco.cc/trials/stevie/), [To Assess the Safety and Efficacy of SP-002 with Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma](https://onco.cc/trials/nct06344052/), [VISMONEO (vismodegib before surgery for locally advanced basal cell carcinoma)](https://onco.cc/trials/vismoneo/)
- key papers: [ERIVANCE BCC: efficacy and safety of vismodegib in advanced basal cell carcinoma](https://onco.cc/key-papers/paper-erivance-vismodegib-sekulic-nejm-2012/), [Vismodegib in recurrent sonic hedgehog-subgroup medulloblastoma (PBTC-025B and PBTC-032)](https://onco.cc/key-papers/paper-robinson-vismodegib-shh-medulloblastoma-jco-2015/)
- pathways: [Basal cell carcinoma (KEGG map)](https://onco.cc/pathways/basal-cell-carcinoma-signalling/), [Fibroblast activation, desmoplasia & matrix stiffness](https://onco.cc/pathways/caf-activation-desmoplasia/), [Proteoglycans in cancer](https://onco.cc/pathways/proteoglycans-in-cancer/)
- drugs: [Patidegib](https://onco.cc/drugs/patidegib/)

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JSON: https://onco.cc/api/v1/entities/vismodegib.json