Drugs / Vismodegib
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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
+72 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 pancreatic neoplasm · 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
+78 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 · Aalborg University Hospital · Aarhus University Hospital · 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 · Herlev 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 · all 84 on the full record

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 79 · started per year

today
US approved 2012
EU approved 2013
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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 05 trials · 2 met primary
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 Met primary
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 Met primary
Phase 117 trials
Phase 1 NCT06616623 Dec 2024 → Dec 2026 non-small cell lung carcinoma Dwight Owen Suspended 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 Stop: Enrollment
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 Mixed
Phase 249 trials · 4 met primary · 6 missed
Phase 2 NCT07802418 Oct 2026 → Jun 2031 expected malignant pancreatic neoplasm Herlev Hospital Not yet recruiting No outcome recorded
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 Stop: Business
Phase 2 NCT03498521 Jul 2018 → Feb 2023 cancer of unknown primary site Hoffmann-La Roche Completed Met primary
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 42 trials · 1 met primary
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 Met primary
Phase not stated1 trial
— NCT01160250 Jul 2010 → Apr 2012 basal cell carcinoma Genentech, Inc. Approved for marketing No outcome recorded
Comparator or background therapy5 trials
Phase 1/2 NCT03158389 Background May 2018 → Feb 2023 adult glioblastoma University Hospital Heidelberg Completed Mixed
Phase 2 NCT06344052 Background Apr 2024 → Sep 2026 overdue 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

News releases announcing trial results or a regulatory action · 5

DateIssuerRelease
2016-11-28 Curis, Inc. Regulatory Curis Announces Full Approval of Roche's Erivedge® in the European Union curis.com ↗
2014-06-23 Curis, Inc. Regulatory Curis Announces Investigational New Drug Application Filing by Roche to Initiate a Phase 2 Study of Erivedge(R) (vismodegib) in Idiopathic Pulmonary Fibrosis curis.com ↗
The IND filing is currently under review by the FDA and we look forward to providing additional details on the study as they become available.
2013-07-15 Curis, Inc. Regulatory Curis Announces the Conditional Approval of Erivedge(R) in the European Union curis.com ↗
This conditional approval makes Erivedge the first licensed treatment in Europe for patients with advanced BCC, a rare form of skin cancer which can be disfiguring, debilitating and even fatal.
2013-05-09 Curis, Inc. Regulatory Curis Announces Approval of Erivedge(R) in Australia curis.com ↗
Erivedge is the first licensed treatment in Australia for patients with advanced basal cell carcinoma (BCC), a rare form of skin cancer which can be disfiguring, debilitating and even fatal.
2013-04-26 Curis, Inc. Regulatory Curis Announces Erivedge(R) Receives Positive CHMP Opinion for Conditional Approval in European Union curis.com ↗
We are extremely pleased that the CHMP has recommended the conditional approval of Erivedge in the EU and we hope that this important medicine will soon be available to patients in Europe,

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 86 sources stand behind the page.

FieldValueCited text
Known as Vismodegib “Axitinib Crizotinib Dabrafenib Trametinib Erlotinib Larotrectinib Olaparib Pembrolizumab Pemigatinib Pertuzumab and trastuzumab (Phesgo9 Selpercatinib Vismodegib” NCT07802418 ↗
77

“Vismodegib in neoadjuvant treatment of locally advanced basal cell carcinoma:” PMID 33997740 ↗ Apr 2021

“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 ↗

NCT01163084 ↗

NCT06357988 ↗

NCT01898598 ↗

NCT00939484 ↗

NCT00982592 ↗

NCT03498521 ↗

Show 67 more sources

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 ↗

NCT01096732 ↗

NCT01173536 ↗

NCT00957229 ↗

NCT00968981 ↗

NCT00991718 ↗

Show 1 more source

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 SMO” PMID 19726763 ↗ Sep 2009