{"entity":{"id":"vismodegib","kind":"drug","name":"Vismodegib","aka":[],"tldr":"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.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vismodegib","links":[{"label":"ERIVANCE (NEJM 2012)","url":"https://doi.org/10.1056/NEJMoa1113713"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vismodegib"}],"tags":["gap-fill"],"related":[],"cancers":["basal-cell-carcinoma","medulloblastoma","medulloblastoma-shh","locally-advanced-bcc"],"sections":[],"technologies":["kinase-inhibitors","hedgehog-inhibitors"],"targets":["smoothened"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["hedgehog-inhibitor-tolerability"],"trials":["nct06344052","stevie","vismoneo"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Erivedge","modality":"Small-molecule smoothened (hedgehog) inhibitor","mechanism":"Binds and inhibits SMO, preventing GLI-mediated transcription downstream of PTCH1 loss.","approvals":[{"region":"US","year":2012,"indication":"Metastatic BCC or locally advanced BCC recurrent after surgery or not amenable to surgery/radiation"},{"region":"EU","year":2013,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/vismodegib/","neighbours":{"cancer":[{"id":"basal-cell-carcinoma","kind":"cancer","name":"Basal cell carcinoma","route":"/cancers/basal-cell-carcinoma/"},{"id":"locally-advanced-bcc","kind":"cancer","name":"Locally advanced and metastatic basal cell carcinoma","route":"/cancers/locally-advanced-bcc/"},{"id":"medulloblastoma","kind":"cancer","name":"Medulloblastoma","route":"/cancers/medulloblastoma/"},{"id":"medulloblastoma-shh","kind":"cancer","name":"SHH-activated medulloblastoma","route":"/cancers/medulloblastoma-shh/"},{"id":"skin-cancer","kind":"cancer","name":"Skin cancer (all types)","route":"/cancers/skin-cancer/"}],"technology":[{"id":"hedgehog-inhibitors","kind":"technology","name":"Hedgehog pathway inhibitors","route":"/technologies/hedgehog-inhibitors/"},{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"ptch1","kind":"target","name":"PTCH1 (Patched 1)","route":"/targets/ptch1/"},{"id":"smoothened","kind":"target","name":"Smoothened (hedgehog pathway)","route":"/targets/smoothened/"}],"company":[{"id":"roche-genentech","kind":"company","name":"Roche / Genentech","route":"/companies/roche-genentech/"}],"term":[{"id":"cancer-stem-cell-theory","kind":"term","name":"Cancer stem cell theory and phenotypic plasticity","route":"/terms/cancer-stem-cell-theory/"},{"id":"medulloblastoma-molecular-groups","kind":"term","name":"Medulloblastoma molecular groups (WNT, SHH, group 3, group 4)","route":"/terms/medulloblastoma-molecular-groups/"},{"id":"hedgehog-inhibitor-tolerability","kind":"term","name":"Why people stop taking hedgehog inhibitors","route":"/terms/hedgehog-inhibitor-tolerability/"}],"trial":[{"id":"cupisco","kind":"trial","name":"CUPISCO","route":"/trials/cupisco/"},{"id":"determine-trial","kind":"trial","name":"DETERMINE","route":"/trials/determine-trial/"},{"id":"erivance","kind":"trial","name":"ERIVANCE BCC","route":"/trials/erivance/"},{"id":"mypathway","kind":"trial","name":"MyPathway","route":"/trials/mypathway/"},{"id":"sjmb12","kind":"trial","name":"SJMB12","route":"/trials/sjmb12/"},{"id":"stevie","kind":"trial","name":"STEVIE (vismodegib in ordinary practice)","route":"/trials/stevie/"},{"id":"nct06344052","kind":"trial","name":"To Assess the Safety and Efficacy of SP-002 with Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma","route":"/trials/nct06344052/"},{"id":"vismoneo","kind":"trial","name":"VISMONEO (vismodegib before surgery for locally advanced basal cell carcinoma)","route":"/trials/vismoneo/"}],"paper":[{"id":"paper-erivance-vismodegib-sekulic-nejm-2012","kind":"paper","name":"ERIVANCE BCC: efficacy and safety of vismodegib in advanced basal cell carcinoma","route":"/key-papers/paper-erivance-vismodegib-sekulic-nejm-2012/"},{"id":"paper-robinson-vismodegib-shh-medulloblastoma-jco-2015","kind":"paper","name":"Vismodegib in recurrent sonic hedgehog-subgroup medulloblastoma (PBTC-025B and PBTC-032)","route":"/key-papers/paper-robinson-vismodegib-shh-medulloblastoma-jco-2015/"}],"pathway":[{"id":"basal-cell-carcinoma-signalling","kind":"pathway","name":"Basal cell carcinoma (KEGG map)","route":"/pathways/basal-cell-carcinoma-signalling/"},{"id":"caf-activation-desmoplasia","kind":"pathway","name":"Fibroblast activation, desmoplasia & matrix stiffness","route":"/pathways/caf-activation-desmoplasia/"},{"id":"proteoglycans-in-cancer","kind":"pathway","name":"Proteoglycans in cancer","route":"/pathways/proteoglycans-in-cancer/"}],"drug":[{"id":"patidegib","kind":"drug","name":"Patidegib","route":"/drugs/patidegib/"}]}}