Structure of Eliglustat
CAS No.: 491833-29-5
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The BI-3802 was designed by Boehringer Ingelheim and could be obtained free of charge through the Boehringer Ingelheim open innovation portal opnMe.com, associated with its negative control.
Eliglustat is a specific and potent inhibitor of glucosylceramide synthase.
Synonyms: Genz 99067; GENZ-112638
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CAS No. : | 491833-29-5 |
Formula : | C23H36N2O4 |
M.W : | 404.54 |
SMILES Code : | CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](C2=CC=C(OCCO3)C3=C2)O)=O |
Synonyms : |
Genz 99067; GENZ-112638
|
MDL No. : | MFCD19443735 |
InChI Key : | FJZZPCZKBUKGGU-AUSIDOKSSA-N |
Pubchem ID : | 23652731 |
GHS Pictogram: |
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Signal Word: | Warning |
Hazard Statements: | H302-H315-H319-H335 |
Precautionary Statements: | P261-P305+P351+P338 |
In Vitro:
Concentration | Treated Time | Description | References |
Madin-Darby canine kidney (MDCK) cells | 1 μM | 1 hour | To assess the inhibitory activity of compounds against glucosylceramide synthase | PMC7919060 |
LP-1 cells | 30 μM | 24 hours | To evaluate the effect of Eliglustat on cells lacking P-glycoprotein expression. Results showed that Eliglustat alone at high concentration reduced cell viability but did not show synergy with proteasome inhibitors. | PMC8033612 |
KMS-18 cells | 30 μM | 24 hours | To evaluate the effect of Eliglustat on P-glycoprotein activity and its synergy with proteasome inhibitors. Results showed that Eliglustat at high concentration (30 μM) synergized with proteasome inhibitors to increase cell death but did not significantly inhibit P-glycoprotein activity. | PMC8033612 |
WT-MDCKII and MDR1-MDCKII cells | 0, 1, 3, 10, 30, 100, 300 nM | 24 hours | Evaluate the activity of GCS inhibitors in intact cells | PMC3269155 |
SF8628 cells | 5 µM, 20 µM, 40 µM | 24 hours | To assess the effect of Eliglustat on GD2 expression. 40 µM Eliglustat significantly reduced the percentage of GD2-positive cells and GD2 mean intensity. | PMC10298524 |
Human blood outgrowth endothelial cells (BOECs) | 8.0 nM | 24 hours | To investigate the effect of Eliglustat on Stx-B binding to the endothelial cell surface. Results showed that Eliglustat significantly reduced Stx-B binding to the cell surface. | PMC8537462 |
Primary human glomerular microvascular endothelial cells (HGMVECs) | 8.0 nM | 24 hours | To investigate the effect of Eliglustat on Stx-B binding to the endothelial cell surface. Results showed that Eliglustat significantly reduced Stx-B binding to the cell surface. | PMC8537462 |
EA.hy926 human endothelial cell line | 200 nM | 3 days | To study the effect of eliglustat on VWF secretion, it was found that eliglustat treatment slightly but significantly increased VWF secretion from both CR-WT and CR-GLA cells. | PMC6320285 |
PC-3M | 128 µM | 48 hours | To evaluate the effect of Eliglustat on prostate cancer cell viability, showing dose-dependent cytotoxicity | PMC7453025 |
RM-9 | 128 µM | 48 hours | To evaluate the effect of Eliglustat on prostate cancer cell viability, showing dose-dependent cytotoxicity | PMC7453025 |
RAW264.7 cells | 0.1, 1, 10, 25, 50 μM | 5 days | Eliglustat inhibited TRAP-positive OC formation in a dose-dependent manner | PMC9780346 |
PC-3M | 128 µM | 6 hours | To evaluate the effect of Eliglustat on lipid metabolism, showing accumulation of intracellular ceramides and reductions in glycosphingolipids | PMC7453025 |
SF8628 cells | 100 µM | 72 hours | To assess the effect of Miglustat on GSL expression by mass spectrometry. 100 µM Miglustat significantly reduced the expression of five gangliosides (GM3, GM2, GM1, GD3, GD2) and neutral GSL. | PMC10298524 |
SF8628 cells | 100 µM | 72 hours | To assess the effect of Miglustat on GD2 expression. 100 µM Miglustat slightly but significantly reduced the percentage of GD2-positive cells and GD2 intensity after 72 hours. | PMC10298524 |
SF8628 cells | 5 µM | 72 hours | To assess the long-term effect of Eliglustat on GD2 expression. 5 µM Eliglustat significantly reduced the percentage of GD2-positive cells and GD2 intensity after 72 hours. | PMC10298524 |
SW480 cells | 100 nmol | Inhibited UGCG-mediated ceramide glycosylation, reversed ECHS1-induced suppression of PI3K/Akt/mTOR pathway and expression of autophagy-related proteins | PMC8494735 | |
HCT116 cells | 100 nmol | Inhibited UGCG-mediated ceramide glycosylation, reversed ECHS1-induced activation of PI3K/Akt/mTOR pathway and expression of autophagy-related proteins | PMC8494735 |
In Vivo:
Administration | Dosage | Frequency | Description | References |
Rats | Sprague Dawley rats | Oral | 10 mg/kg | Single dose | To investigate the effects of amiodarone and quinidine on the metabolism of Eliglustat. Results showed that amiodarone and quinidine significantly increased the main pharmacokinetic parameters (AUC0→t, AUC0→∞, and Cmax) of eliglustat and decreased clearance obviously. | PMC6913762 |
Mice | C57BL/6 mice | Intraperitoneal injection | 10 mg/kg | Once daily for 3 days | To evaluate the effect of the compound on brain glucosylceramide levels | PMC7919060 |
C57BL/6 mice | Wild-type mice | Intraperitoneal injection | 10 mg/kg/day | Once daily for 3 days | Evaluate the effect of 3h on brain GlcCer levels | PMC3269155 |
C57BL/6J mice | Healthy mice | Chow | 150 mg/kg/day | 19 days | Eliglustat increased trabecular bone volume by inhibiting OCs | PMC9780346 |
C57BL/6N mice | RM-9-luciferase prostate cancer model | Intraperitoneal injection | 60 mg/kg | Once daily for 15 days | To evaluate the effect of Eliglustat on prostate cancer tumor growth, showing suppression of tumor growth | PMC7453025 |
BALB/c nude mice | Subcutaneous tumor model | Intraperitoneal injection | 60 mg/kg | Daily for four weeks | Reversed ECHS1-induced oxaliplatin resistance and subcutaneous tumor growth | PMC8494735 |
Clinical Trial:
NCT Number | Conditions | Phases | Recruitment | Completion Date | Locations |
NCT01074944 | Gaucher Disease | Phase 3 | Completed | - | - |
NCT00358150 | Gaucher Disease, Type 1 ... More >> Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form Less << | Phase 2 | Completed | - | United States, New York ... More >> New York University New York, New York, United States New York, New York, United States Argentina Aprillus Asistencia e Investigación Buenos Aires, Argentina Hospital de Oncologia Maria Curie Buenos Aires, Argentina IMAI Buenos Aires, Argentina Instituto Argentino de Diagnostico y Tratamiento (IADT) Buenos Aires, Argentina Buenos Aires, Argentina Hospital Ramos Mejia Ciudad Autonoma de Buenos Aires, Argentina Israel Rambam Medical Center Haifa, Israel Haifa, Israel Sha'are Zedek Medical Centre Jerusalem, Israel Jerusalem, Israel Italy Universita degli Studi di Milano Milano, Italy Mexico Instituto Mexicano del Seguro Social D.f., Mexico Mexico City, Mexico Russian Federation Hematology Research Center of Ministry of Healthcare of the Russian Federation Moscow, Russian Federation Moscow, Russian Federation Less << |
NCT00943111 | - | Completed | - | - | |
NCT01074944 | - | Completed | - | - | |
NCT00891202 | - | Completed | - | - | |
NCT00891202 | Gaucher Disease, Type 1 | Phase 3 | Completed | - | United States, California ... More >> UCSF MS Center San Francisco, California, United States, 94143 United States, Connecticut Yale University School of Medicine New Haven, Connecticut, United States, 06510 United States, Georgia Emory University Medical Genetics Decatur, Georgia, United States, 30033 United States, Kansas University of Kansas Medical Center, Division of Hematology/Oncology, Dept. of Medicine Westwood, Kansas, United States, 66160 United States, New York New York University School of Medicine, Neurology Department New York, New York, United States, 10016 Mount Sinai School of Medicine New York, New York, United States, 10029 Bulgaria University hospital "Alexandrovska" Sofia Sofia, Bulgaria, 1431 Canada Sir Mortimer B. Davis - Jewish General Hospital Montreal, Quebec, Canada, H3T 1E2 Mount Sinai Hospital and the Samuel Lunenfeld Research Institute Toronto Ontario, Canada, M5G 1X5 Colombia Hospital de San Jose Bogota, Colombia India Christian Medical College Hospital Vellore, India, 632004 Israel Rabin Medical Center, Beilinson Hospital Petach Tikvah, Israel, 49100 Lebanon Hôtel-Dieu de France University Hospital Beirut, Lebanon Mexico OCA Hospital Monterrey, Nuevo Leon, Mexico Russian Federation Hematology Research Center of Ministry of Healthcare of the Russian Federation Moscow, Russian Federation, 125167 Serbia Institut za endokrinologiju Belgrade, Serbia, 11000 Tunisia Hopital La-Rabta Tunis, TN, Tunisia, 1007 United Kingdom Royal Free Hospital London, United Kingdom, NW3 2QG Less << |
NCT02536937 | Gaucher Disease | Phase 1 | Completed | - | United States, Florida ... More >> Investigational Site Number 840004 Miami, Florida, United States, 33014 United States, Minnesota Investigational Site Number 840002 St. Paul, Minnesota, United States, 55144 United States, Tennessee Investigational Site Number 840001 Knoxville, Tennessee, United States, 37920 Less << |
NCT00943111 | Gaucher Disease, Type 1 | Phase 3 | Completed | - | - |
NCT02422654 | Gaucher Disease | Phase 1 | Completed | - | United States, Indiana ... More >> Investigational Site Number 840001 Evansville, Indiana, United States, 47710 Less << |
NCT02536911 | Gaucher Disease | Phase 1 | Completed | - | United States, Florida ... More >> Investigational Site Number 840002 Miami, Florida, United States, 33014 United States, Tennessee Investigational Site Number 840001 Knoxville, Tennessee, United States, 37920 Less << |
NCT01659944 | Healthy | Phase 1 | Completed | - | United States, Texas ... More >> Austin, Texas, United States Less << |
NCT01357811 | Healthy Volunteer | Phase 1 | Completed | - | United States, Texas ... More >> PPD Phase I Clinic Austin, Texas, United States Less << |
NCT00358150 | - | Completed | - | - | |
NCT01452542 | Healthy Volunteer | Phase 1 | Completed | - | United States, Texas ... More >> PPD Phase 1 Unit Austin, Texas, United States Less << |
NCT02536755 | Gaucher's Disease | Phase 3 | Enrolling by invitation | June 22, 2021 | Canada ... More >> Investigational Site Number 124002 Montreal, Canada, H3T 1E2 Russian Federation Investigational Site Number 643001 Moscow, Russian Federation, 125167 Investigational Site Number 643002 St-Petersburg, Russian Federation, 197341 Less << |
NCT03519646 | Gaucher Disease, Type III | COMPLETED | 2020-09-11 | National Taiwan University Hos... More >>pital, Taipei, 10041, Taiwan Less << | |
NCT03485677 | Gaucher's Disease Type I ... More >> Gaucher's Disease Type III Less << | Phase 3 | Recruiting | March 2023 | Canada ... More >> Investigational Site Number 1240002 Recruiting Calgary, Canada, T3B 6A8 Investigational Site Number 1240001 Recruiting Toronto, Canada, M5G 1X8 Investigational Site Number 1240003 Recruiting Vancouver, Canada, V6H 3V4 Italy Investigational Site Number 3800001 Recruiting Genova, Italy, 16147 Russian Federation Investigational Site Number 6430001 Recruiting Moscow, Russian Federation, 119049 Investigational Site Number 6430004 Recruiting Moscow, Russian Federation, 119991 Investigational Site Number 6430005 Recruiting St-Petersburg, Russian Federation, 197341 Investigational Site Number 6430002 Recruiting Tomsk, Russian Federation, 634050 Turkey Investigational Site Number 7920001 Recruiting Ankara, Turkey, 06500 Investigational Site Number 7920002 Recruiting Izmir, Turkey, 35040 Less << |
NCT06193304 | Gaucher's Disease | PHASE1 | COMPLETED | 2014-09-21 | Covance Clinical Researsh Unit... More >>, Evansville, Indiana, 47710, United States Less << |
NCT06188325 | Gaucher's Disease | PHASE1 | COMPLETED | 2018-03-26 | M.D.Covance Clinical Research ... More >>Unit 1341 W, Dallas, Texas, 75247, United States Less << |
Bio Calculators | ||||
Preparing Stock Solutions | ![]() |
1mg | 5mg | 10mg |
1 mM 5 mM 10 mM |
2.47mL 0.49mL 0.25mL |
12.36mL 2.47mL 1.24mL |
24.72mL 4.94mL 2.47mL |
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Dissolving Methods |
in order to ensure the reliability of the experimental results, the clarified stock solution can be properly preserved according to the storage conditions; The working fluid for in vivo experiment is recommended to be prepared now and used on the same day; The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound. Please choose the appropriate dissolution scheme according to your animal administration guide.For the following dissolution schemes, clear stock solution should be prepared according to in vitro experiments, and then cosolvent should be added in turn:
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