Drugs / Sonidegib
last change Sep 2025 re-read 2 minutes ago

Sonidegib

Small molecule targets SMO via inhibition

Developed for
basal cell carcinoma · medulloblastoma · skin basal cell carcinoma · acquired polycythemia vera · essential thrombocythemia · hepatocellular carcinoma · malignant pancreatic neoplasm · ovarian cancer
+40 more · pancreatic ductal adenocarcinoma · plasma cell myeloma · primary myelofibrosis · acute leukemia · astrocytoma (excluding glioblastoma) · breast cancer · breast neoplasm · childhood acute myeloid leukemia · chronic graft versus host disease · chronic myelomonocytic leukemia · cirrhosis of liver · colorectal cancer · cutaneous melanoma · esophageal cancer · gastric adenocarcinoma · gastric cancer · gastroesophageal cancer · gastroesophageal junction adenocarcinoma · glioblastoma · head and neck squamous cell carcinoma · hepatoblastoma · hormone receptor-positive breast cancer · invasive carcinoma · lung cancer · medulloblastoma SHH activated and TP53 mutant · medulloblastoma SHH activated and TP53 wild-type · metastatic malignant neoplasm · metastatic melanoma · myelodysplastic syndrome · neuroblastoma · nevoid basal cell carcinoma syndrome · non-small cell lung carcinoma · pancreatic adenocarcinoma · pancreatic neoplasm · Philadelphia-positive myelogenous leukemia · prostate cancer · rhabdomyosarcoma · skin cancer · triple-negative breast carcinoma · urothelial carcinoma
Investigated by
Novartis Pharmaceuticals · Novartis · National Cancer Institute (NCI) · Mayo Clinic · Sun Pharmaceutical Industries, Inc. · Massachusetts General Hospital
+26 more · Anne Chang · Dana-Farber Cancer Institute · European Organisation for Research and Treatment of Cancer - EORTC · Gruppo Oncologico del Nord-Ovest · Jason K. Sicklick, M.D. · Jemincare · M.D. Anderson Cancer Center · Martin Gutierrez · Melanoma Institute Australia · Memorial Sloan Kettering Cancer Center · Nathalie Zeitouni · Reinhard Dummer · Rutgers Cancer Institute of New Jersey · Rutgers, The State University of New Jersey · SCRI Development Innovations, LLC · Sanofi · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Spanish Breast Cancer Research Group · St. Jude Children's Research Hospital · Swiss Cancer Institute · The Skip Viragh Foundation · University of Alabama at Birmingham · University of Florida · University of Utah · Washington University School of Medicine · Zhejiang Hangyu Pharmaceutical Co., Ltd.

Regulatory milestones approvals, filings & regulatory actions · 3 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) ODOMZO 2017-09-18 fda.gov
Approved EU (EMA) Odomzo 2015-08-14 europa.eu
Approved US (FDA) ODOMZO 2015-07-24 fda.gov

Trials 40 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT01694589 Nov 2012 → Jun 2014 pancreatic neoplasm Rutgers, The State University of New Jersey Withdrawn No outcome recorded
Phase 120 trials
Phase 1 NCT06623201 Oct 2024 → Dec 2026 expected basal cell carcinoma Nathalie Zeitouni Recruiting No outcome recorded
Phase 1 NCT04007744 Feb 2020 → May 2025 overdue cutaneous melanoma, gastric adenocarcinoma, gastroesophageal junction adenocarcinoma, head and neck squamous cell carcinoma +4 Mayo Clinic Active not recruiting No outcome recorded
Phase 1 NCT03434262 Mar 2018 → Sep 2022 IDH-wildtype glioblastoma, RELA fusion-positive ependymoma, anaplastic ependymoma, anaplastic ganglioglioma +19 St. Jude Children's Research Hospital Completed No outcome recorded
Phase 1 NCT02138929 Nov 2014 → Jun 2020 esophageal cancer M.D. Anderson Cancer Center Completed No outcome recorded
Phase 1 NCT02195973 Sep 2014 → Dec 2016 ovarian cancer University of Alabama at Birmingham Completed No outcome recorded
Phase 1 NCT02182622 Jul 2014 → Dec 2015 prostate cancer Martin Gutierrez Withdrawn No outcome recorded
Phase 1 NCT02151864 Jul 2014 → Dec 2016 cirrhosis of liver, hepatocellular carcinoma Jason K. Sicklick, M.D. Completed No outcome recorded
Phase 1 NCT02129101 May 2014 → Dec 2016 acquired polycythemia vera, childhood acute myeloid leukemia, chronic myelomonocytic leukemia, essential thrombocythemia +2 Mayo Clinic Completed No outcome recorded
Phase 1 NCT02027376 May 2014 → Apr 2016 breast cancer Spanish Breast Cancer Research Group Completed No outcome recorded
Phase 1 NCT02086513 Apr 2014 → Dec 2016 chronic graft versus host disease Massachusetts General Hospital Terminated No outcome recorded
Phase 1/23 trials
Phase 1/2 NCT01787552 May 2013 → Apr 2018 acquired polycythemia vera, blood coagulation disease, essential thrombocythemia, hemorrhagic disease +1 Novartis Pharmaceuticals Completed No outcome recorded
Phase 1/2 NCT01431794 Dec 2011 → Nov 2018 pancreatic ductal adenocarcinoma Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Terminated No outcome recorded
Phase 1/2 NCT01125800 Feb 2011 → Oct 2014 astrocytoma (excluding glioblastoma), hepatoblastoma, medulloblastoma, neuroblastoma +1 Novartis Pharmaceuticals Completed No outcome recorded
Phase 214 trials
Phase 2 NCT05669339 Dec 2024 → Apr 2027 expected hepatocellular carcinoma University of Florida Recruiting No outcome recorded
Phase 2 NCT04402073 Nov 2022 → Jun 2025 medulloblastoma European Organisation for Research and Treatment of Cancer - EORTC Terminated No outcome recorded
Phase 2 NCT04679480 Feb 2021 → Dec 2024 overdue basal cell carcinoma Reinhard Dummer Active not recruiting No outcome recorded
Phase 2 NCT04806646 Jan 2021 → Dec 2024 overdue skin basal cell carcinoma Gruppo Oncologico del Nord-Ovest Active not recruiting No outcome recorded
Phase 2 NCT03534947 Jul 2019 → Mar 2025 invasive carcinoma, skin basal cell carcinoma Melanoma Institute Australia Completed No outcome recorded
Phase 2 NCT02303041 Feb 2015 → Apr 2017 basal cell carcinoma, nevoid basal cell carcinoma syndrome, skin cancer Anne Chang Terminated No outcome recorded
Phase 2 NCT02254551 Jan → Oct 2015 plasma cell myeloma SCRI Development Innovations, LLC Terminated No outcome recorded
Phase 2 NCT01757327 Apr 2014 → Dec 2016 breast neoplasm Washington University School of Medicine Withdrawn No outcome recorded
Phase 2 NCT02002689 Feb 2014 → Mar 2015 Novartis Pharmaceuticals Terminated No outcome recorded
Phase 2 NCT02086552 Jan 2014 → Jan 2018 plasma cell myeloma Mayo Clinic Completed No outcome recorded
Phase 41 trial
Phase 4 NCT06880848 Jun 2023 → Aug 2025 basal cell carcinoma Jemincare Completed No outcome recorded
Phase not applicable1 trial
NCT01911416 Jan → Oct 2014 pancreatic ductal adenocarcinoma University of Utah Withdrawn No outcome recorded
Also used as a comparator or background therapy in 4 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT02111187 Comparator Apr 2014 → Jan 2017 prostate cancer Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Completed No outcome recorded
Phase 22 trials
Phase 2 NCT01350115 Comparator Apr 2011 → Oct 2012 basal cell carcinoma, nevoid basal cell carcinoma syndrome Novartis Pharmaceuticals Completed No outcome recorded
Phase 2 NCT00961896 Comparator Jul 2009 → Aug 2010 skin basal cell carcinoma Novartis Pharmaceuticals Completed No outcome recorded
Phase not applicable1 trial
NCT01529450 Comparator Feb 2012 → Aug 2013 basal cell carcinoma Anne Chang Completed No outcome recorded

Press releases naming this drug 2 releases

DateIssuerRelease
2015-08-20 Novartis Pharmaceuticals Novartis drug Odomzo® gains EU approval for locally advanced basal cell carcinoma, providing new non-invasive therapy for patients novartis.com
2015-07-24 Novartis Pharmaceuticals FDA approves Novartis drug Odomzo® (sonidegib) for locally advanced basal cell carcinoma (laBCC), a form of skin cancer novartis.com

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 47 sources stand behind the page.

FieldValueCited text
Known as sonidegib Sonidegib (Odomzo) is a compound that was approved by the US Food and Drug Administration in July 2015 as a treatment option for BCC.” NCT06623201
16

“The effect of sonidegib (LDE225) on the pharmacokinetics of bupropion and warfarin in patients with advanced solid tumours.” PMID 32736411 Aug 2020

“Safety and efficacy of the combination of sonidegib and ruxolitinib in myelofibrosis: a phase 1b/2 dose-finding study.” PMID 32634234 Jul 2020

“Phase I, multicenter, open-label, dose-escalation study of sonidegib in Asian patients with advanced solid tumors.” PMID 27467121 Sep 2016

“The Hedgehog Inhibitor used for the trial will be Sonidegib.” NCT04679480

“the smoothened (SMO) inhibitor, sonidegib (LDE225)” PMID 26546616 Nov 2015

Sonidegib (LDE225)” NCT01708174

NCT02303041

NCT04806646

NCT05669339

NCT06880848

NCT03534947

NCT03434262

NCT02086552

NCT04402073

NCT02129101

NCT04007744

Known as Erismodegib ClinicalTrials.gov intervention name — accepted as the source's own label NCT01757327
Known as Erismodegib phosphate ChEMBL registry synonym — accepted as the source's own label CHEMBL2105737
Known as LDE225 “Sonidegib (LDE225)” NCT01708174
28

NCT02182622

NCT01327053

NCT00880308

NCT01954355

NCT01033019

NCT02111187

NCT02195973

NCT01911416

NCT02138929

NCT00961896

NCT02151864

NCT01694589

NCT01456676

NCT02002689

NCT01826214

NCT01529450

NCT01579929

NCT01764776

NCT02027376

NCT01487785

NCT02086513

NCT01769768

NCT01350115

NCT02254551

NCT01576666

NCT01431794

NCT01485744

NCT01125800

Known as NVP-LDE 225 ChEMBL registry synonym — accepted as the source's own label CHEMBL2105737
Known as Odomzo “Sonidegib (Odomzo) is a compound that was approved by the US Food and Drug Administration in July 2015 as a treatment option for BCC.” NCT06623201
Known as Sonidegib (LDE225) Sonidegib (LDE225)NCT02195973
Known as Sonidegib diphosphate ChEMBL registry synonym — accepted as the source's own label CHEMBL2105737
Known as SONIDEGIB PHOSPHATE ChEMBL registry synonym — accepted as the source's own label CHEMBL2105737
Action Inhibit “LDE225 (a specific Smoothened inhibitor)” NCT00961896
Modality Small molecule CHEMBL2105737
Route Oral “LDE225 given orally” NCT00880308
Target SMO smoothened (SMO) inhibitor” PMID 26546616 Nov 2015