Drugs / Vismodegib
last change Jul 2026 re-read 2 minutes ago

Vismodegib

also known as GDC-0449 · ERIVEDGE · NSC-747691 · NSC-755986

Small molecule targets GLI1, SMO via inhibition

Developed for
basal cell carcinoma · cancer · pancreatic ductal adenocarcinoma · plasma cell myeloma · skin basal cell carcinoma · childhood medulloblastoma · pancreatic adenocarcinoma · acute myeloid leukemia
+71 more · cancer of unknown primary site · chronic lymphocytic leukemia/small lymphocytic lymphoma · colorectal cancer · female breast carcinoma · idiopathic pulmonary fibrosis · metastatic malignant neoplasm · myelodysplastic syndrome · nevoid basal cell carcinoma syndrome · ovarian cancer · ovarian carcinoma · pancreatic acinar cell carcinoma · prostate carcinoma · refractory malignant neoplasm · urinary bladder cancer · adult epithelioid sarcoma · adult extraskeletal osteosarcoma · adult fibrosarcoma · adult glioblastoma · adult liposarcoma · adult lymphoma · adult malignant mesenchymoma · adult medulloblastoma · alveolar soft part sarcoma · biliary tract cancer · brain stem glioma · breast cancer · chondrosarcoma · chronic graft versus host disease · clear cell chondrosarcoma · clear cell sarcoma of kidney · dedifferentiated chondrosarcoma · dermatofibrosarcoma protuberans · desmoplastic small round cell tumor · diffuse large B-cell lymphoma · digestive system carcinoma · epithelioid hemangioendothelioma · Ewing sarcoma/peripheral primitive neuroectodermal tumor · extraskeletal myxoid chondrosarcoma · fallopian tube carcinoma · gastric adenocarcinoma · gastric neoplasm · gastroesophageal junction adenocarcinoma · gastrointestinal stromal tumor · hematopoietic and lymphoid cell neoplasm · intracranial meningioma · Kaposi's sarcoma · Keratocystic odontogenic tumor · malignant peripheral nerve sheath tumor · medulloblastoma SHH activated · mesenchymal chondrosarcoma · myelofibrosis · non-Hodgkin lymphoma · non-small cell lung carcinoma · ovarian sarcoma · pancreas disorder · pancreatic large cell neuroendocrine carcinoma · primary central nervous system lymphoma · primary peritoneal carcinoma · prostate adenocarcinoma · prostate cancer · rhabdomyosarcoma · salivary gland cancer · small cell lung carcinoma · small intestine leiomyosarcoma · soft tissue sarcoma · synovial sarcoma · triple-negative breast carcinoma · undifferentiated pleomorphic sarcoma · urothelial carcinoma · uterine corpus sarcoma · brain neoplasm
Investigated by
Genentech, Inc. · National Cancer Institute (NCI) · Hoffmann-La Roche · Roche Pharma AG · GlaxoSmithKline · Pfizer
+74 more · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · AstraZeneca · Bristol-Myers Squibb · Mayo Clinic · Merck Sharp & Dohme LLC · OHSU Knight Cancer Institute · Oregon Health and Science University · University of Arizona · Alliance for Clinical Trials in Oncology · Amgen · Anne Chang · Bayer · Boehringer Ingelheim · Brain Science Foundation · Canadian Cancer Trials Group · Celgene Corporation · Centre Leon Berard · Clinica Universidad de Navarra, Universidad de Navarra · Clovis Oncology - Pharma and · Columbia University · Dutch Cancer Society · Dwight Owen · Eisai Inc. · Eli Lilly and Company · Foundation Medicine · Giselle Sholler · H. Lee Moffitt Cancer Center and Research Institute · Haukeland University Hospital · Helse Fonna · Helse Forde · Helse Møre og Romsdal HF · Helse Nord-Trøndelag HF · Helse Stavanger HF · Helsinki University Central Hospital · Hospital of Southern Norway Trust · Incyte Corporation · Innlandet Hospital Trust · Ipsen · Istituto Clinico Humanitas · Janssen, LP · Jean-Luc Van Laethem · Lisa Bax · Lovisenberg Diakonale Hospital · Ministry of Health, France · NYU College of Dentistry · Nordland Hospital · Novartis · Oslo University Hospital · Ostfold Hospital Trust · Phoenix Children's Hospital · Ronald Buckanovich · SRH Wald-Klinikum Gera GmbH · Samsung Medical Center · Sarit Assouline · Seagen Inc. · St. Jude Children's Research Hospital · St. Olavs Hospital · Stand Up To Cancer · Stanford University · Stelvio for Life · Sue Yom · Sykehuset Telemark · The Hospital of Vestfold · The Lymphoma Academic Research Organisation · The Netherlands Cancer Institute · UCSF Benioff Children's Hospital Oakland · Ulrik Lassen · University Hospital of North Norway · University Hospital, Akershus · University Hospital, Bordeaux · University Hospital, Lille · University Hospitals Cleveland Medical Center · University of Michigan Rogel Cancer Center · Vestre Viken Hospital Trust

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) ERIVEDGE 2019-01-18 fda.gov
Approved EU (EMA) Erivedge 2013-07-12 europa.eu
Refused (EMA) EU (EMA) Erivedge 2013-04-26 europa.eu
Approved US (FDA) ERIVEDGE 2012-01-30 fda.gov

Trials 78 · started per year

today
US approved 2012
EU approved 2013
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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 05 trials
Phase 0 NCT05651828 Mar 2023 → Dec 2028 expected basal cell carcinoma H. Lee Moffitt Cancer Center and Research Institute Recruiting No outcome recorded
Phase 0 NCT05238831 Jan 2023 → May 2026 female breast carcinoma, ovarian carcinoma, pancreatic ductal adenocarcinoma, pancreatic large cell neuroendocrine carcinoma +2 OHSU Knight Cancer Institute Withdrawn No outcome recorded
Phase 0 NCT02115828 Apr 2014 → Apr 2016 prostate cancer Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Completed No outcome recorded
Phase 0 NCT01713218 Dec 2012 → Jun 2014 pancreatic adenocarcinoma Jean-Luc Van Laethem Unknown No outcome recorded
Phase 0 NCT01631331 Jun 2012 → Sep 2015 skin basal cell carcinoma Stanford University Completed No outcome recorded
Phase 117 trials
Phase 1 NCT06616623 Dec 2024 → Dec 2026 expected non-small cell lung carcinoma Dwight Owen Recruiting No outcome recorded
Phase 1 NCT03878524 Apr → Dec 2020 acute myeloid leukemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, blast phase chronic myelogenous leukemia, BCR-ABL1 positive, castration-resistant prostate carcinoma +14 OHSU Knight Cancer Institute Terminated No outcome recorded
Phase 1 NCT02593760 Jan 2016 → Mar 2017 myelofibrosis Hoffmann-La Roche Completed No outcome recorded
Phase 1 NCT02648048 Jan → Nov 2016 idiopathic pulmonary fibrosis Hoffmann-La Roche Completed No outcome recorded
Phase 1 NCT02639117 Nov 2015 → Nov 2016 basal cell carcinoma University of Arizona Completed No outcome recorded
Phase 1 NCT01772290 Feb → May 2013 Genentech, Inc. Completed No outcome recorded
Phase 1 NCT01537107 Mar 2012 → Jun 2018 pancreatic acinar cell carcinoma, pancreatic ductal adenocarcinoma Mayo Clinic Completed No outcome recorded
Phase 1 NCT01546519 Mar 2012 → Apr 2014 cancer Genentech, Inc. Completed No outcome recorded
Phase 1 NCT01330173 Dec 2010 → Oct 2014 plasma cell myeloma National Cancer Institute (NCI) Completed No outcome recorded
Phase 1 NCT01209143 Nov 2010 → Mar 2012 Genentech, Inc. Completed No outcome recorded
Phase 1/25 trials
Phase 1/2 NCT02690948 Feb 2016 → Nov 2017 skin basal cell carcinoma Anne Chang Completed No outcome recorded
Phase 1/2 NCT01601184 Jun 2012 → Sep 2017 medulloblastoma SHH activated Centre Leon Berard Terminated No outcome recorded
Phase 1/2 NCT01163084 Jul 2010 → Jun 2012 prostate adenocarcinoma National Cancer Institute (NCI) Terminated No outcome recorded
Phase 1/2 NCT01154452 Jun 2010 → Feb 2015 Ewing sarcoma/peripheral primitive neuroectodermal tumor, Kaposi's sarcoma, adult epithelioid sarcoma, adult extraskeletal osteosarcoma +18 National Cancer Institute (NCI) Completed No outcome recorded
Phase 1/2 NCT01174264 Oct 2009 → Dec 2014 cancer National Cancer Institute (NCI) Completed No outcome recorded
Phase 248 trials
Phase 2 NCT05538091 May 2023 → Aug 2031 expected fallopian tube carcinoma, primary peritoneal carcinoma Ronald Buckanovich Recruiting No outcome recorded
Phase 2 NCT05159245 Dec 2021 → Nov 2026 expected cancer, hematopoietic and lymphoid cell neoplasm Helsinki University Central Hospital Recruiting No outcome recorded
Phase 2 NCT04817956 Apr 2021 → Mar 2040 expected metastatic malignant neoplasm Oslo University Hospital Recruiting No outcome recorded
Phase 2 NCT04341181 Aug 2020 → Apr 2029 expected cancer Ulrik Lassen Recruiting No outcome recorded
Phase 2 NCT03767439 Jul → Dec 2019 nevoid basal cell carcinoma syndrome Columbia University Withdrawn No outcome recorded
Phase 2 NCT03498521 Jul 2018 → Feb 2023 cancer of unknown primary site Hoffmann-La Roche Completed No outcome recorded
Phase 2 NCT03297606 Mar 2018 → Dec 2026 expected non-Hodgkin lymphoma, plasma cell myeloma Canadian Cancer Trials Group Recruiting No outcome recorded
Phase 2 NCT02788201 Mar 2017 → Oct 2019 urinary bladder cancer, urothelial carcinoma National Cancer Institute (NCI) Completed No outcome recorded
Phase 2 NCT03052478 Feb 2017 → Dec 2019 gastric neoplasm Samsung Medical Center Completed No outcome recorded
Phase 2 NCT03035188 Jan 2017 → Jan 2020 basal cell carcinoma SRH Wald-Klinikum Gera GmbH Completed No outcome recorded
Phase 42 trials
Phase 4 NCT03610022 Sep 2018 → May 2021 skin basal cell carcinoma University Hospital, Bordeaux Completed No outcome recorded
Phase 4 NCT02436408 Jul 2015 → Sep 2020 basal cell carcinoma University of Michigan Rogel Cancer Center Completed No outcome recorded
Phase not stated1 trial
NCT01160250 Jul 2010 → Apr 2012 basal cell carcinoma Genentech, Inc. Approved for marketing No outcome recorded
Also used as a comparator or background therapy in 5 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/21 trial
Phase 1/2 NCT03158389 Background May 2018 → Feb 2023 adult glioblastoma University Hospital Heidelberg Completed No outcome recorded
Phase 24 trials
Phase 2 NCT06344052 Background Apr 2024 → Sep 2026 expected basal cell carcinoma Stamford Pharmaceuticals, Inc. Recruiting No outcome recorded
Phase 2 NCT05561634 Background Jun 2023 → Jun 2029 expected basal cell carcinoma University Hospital, Lille Recruiting No outcome recorded
Phase 2 NCT01543581 Comparator May 2012 → Jun 2013 basal cell carcinoma Abel Torres, MD Completed No outcome recorded
Phase 2 NCT01556009 Comparator Apr 2012 → Dec 2015 nevoid basal cell carcinoma syndrome UCSF Benioff Children's Hospital Oakland Completed No outcome recorded

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 85 sources stand behind the page.

FieldValueCited text
Known as Vismodegib Vismodegib in neoadjuvant treatment of locally advanced basal cell carcinoma:” PMID 33997740 Apr 2021
76

“Gemcitabine and nab-Paclitaxel in combination with GDC-0449 (Vismodegib)” NCT01088815

vismodegib used outside of indication, based on biomarker” NCT04817956

“Vincristine, Vismodegib, and/or Vorinostat” NCT02788201

NCT02639117

NCT02648048

NCT01154452

NCT05238831

NCT02366312

NCT01631331

NCT05651828

NCT01601184

NCT01201915

NCT01174264

NCT02115828

NCT00887159

NCT02956889

NCT01239316

NCT04341181

NCT02465060

NCT02067104

NCT01160250

NCT01700049

NCT02690948

NCT00739661

NCT02073838

NCT01772290

NCT03878524

NCT01878617

NCT02337517

NCT00980343

NCT01546519

NCT01713218

NCT05538091

NCT02436408

NCT01330173

NCT02523014

NCT06344052

NCT01543581

NCT03767439

NCT01209143

NCT02694224

NCT01071564

NCT03035188

NCT02091141

NCT01267955

NCT01163084

NCT00939484

NCT01944943

NCT03158389

NCT03610022

NCT01774253

NCT03498521

NCT01835626

NCT00822458

NCT00833417

NCT05159245

NCT00959647

NCT02593760

NCT03297606

NCT01195415

NCT05561634

NCT02925234

NCT00878163

NCT01064622

NCT01537107

NCT03052478

NCT02168530

NCT01898598

NCT00982592

NCT06357988

NCT06616623

NCT00636610

NCT01556009

NCT01815840

NCT01880437

NCT01367665

Known as ERIVEDGE “Vismodegib (ERIVEDGE®) at the standard dose of 150 mg/day orally is indicated for the treatment of advanced Basal Cell Carcinoma (BCC) and is associated with many adverse effects.” NCT03610022
1

“GDC-0449, ERIVEDGE®” NCT01944943

Known as GDC-0449 “This single-arm, open-label, multi-center study will evaluate the safety and efficacy of vismodegib (GDC-0449) in patients with locally advanced or metastatic basal cell carcinoma.” NCT01367665
11

“A Study of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) As Maintenance Therapy in Patients With Ovarian Cancer in a Second or Third Complete Remission” NCT00739661

“Vismodegib, (GDC-0449) is an orally delivered small molecule hedgehog pathway inhibitor.” NCT01543581

“vismodegib (GDC-0449)” NCT01160250

GDC-0449, ERIVEDGE®” NCT01944943

NCT00607724

NCT01173536

NCT01088815

NCT00957229

NCT00968981

NCT00991718

NCT01096732

Known as NSC-747691 ChEMBL registry synonym — accepted as the source's own label CHEMBL473417
Known as NSC-755986 ChEMBL registry synonym — accepted as the source's own label CHEMBL473417
Action Inhibit inhibitor of SMO” PMID 19726763 Sep 2009
Modality Small molecule “GDC-0449, a small-molecule inhibitor of SMO” PMID 19726763 Sep 2009
Route Oral “Patients receive oral systemic Hedgehog antagonist GDC-0449” NCT00607724
Target SMO “inhibitor of SMOPMID 19726763 Sep 2009