Malignant solid neoplasm, SMO-mutated or PTCH-1-mutated (Hedgehog)

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1 variants on this page

All lines of therapy

Vismodegib monotherapy


Study Dates of enrollment Evidence
Hainsworth et al. 2018 (MyPathway) 2014-2016 Phase 2a

Biomarker eligibility criteria

  • SMO p.W535L or other nonsynonymous mutations, or PTCH-1 loss-of-function mutations

Targeted therapy

Continued indefinitely


  1. MyPathway: Hainsworth JD, Meric-Bernstam F, Swanton C, Hurwitz H, Spigel DR, Sweeney C, Burris H, Bose R, Yoo B, Stein A, Beattie M, Kurzrock R. Targeted therapy for advanced solid tumors on the basis of molecular profiles: results from MyPathway, an open-label, phase IIa multiple basket study. J Clin Oncol. 2018 Feb 20;36(6):536-542. Epub 2018 Jan 10. link to original article dosing details in manuscript have been reviewed by our editors PubMed NCT02091141