Identification
- Summary
Eliglustat is a glucosylceramide synthase used to treat type 1 Gaucher disease in patients who are CYP2D6 extensive, intermediate, or poor metabolizers.
- Brand Names
- Cerdelga
- Generic Name
- Eliglustat
- DrugBank Accession Number
- DB09039
- Background
Eliglustat is a glucosylceramide synthase inhibitor used for the long-term treatment of type 1 Gaucher disease.2,6 Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide into glucose and ceramide. In patients with Gaucher disease, the accumulation of glucosylceramide leads to the formation of Gaucher cells that infiltrate the liver, spleen, bone marrow and other organs. This leads to complications such as anemia and thrombocytopenia.6,4 By inhibiting glucosylceramide synthase, eliglustat reduces the accumulation of glucosylceramide.6
Eliglustat is mainly metabolized by CYP2D6.6 Patients selected for eliglustat treatment undergo an FDA-cleared genotyping test to establish if they are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs). The results of this test dictate eliglustat dosing recommendations for each type of patient. There are no dosing recommendations for CYP2D6 ultra-rapid or indeterminate metabolizers.6,1 Eliglustat was approved by the FDA in August 2014 as an oral substrate reduction therapy for the first-line treatment of type 1 Gaucher disease.6,1 Enzyme replacement continues to be the standard of care for the treatment of type 1 Gaucher disease (imiglucerase, velaglucerase alfa, taliglucerase alfa); however, oral substrate reduction therapies with favourable safety profiles, such as eliglustat, represent a treatment alternative.5,1
- Type
- Small Molecule
- Groups
- Approved, Investigational
- Structure
Structure for Eliglustat (DB09039)
- Weight
- Average: 404.551
Monoisotopic: 404.267507647 - Chemical Formula
- C23H36N2O4
- Synonyms
- Eliglustat
- éliglustat
- Eliglustatum
- N-[(1R,2R)-1-(2,3-Dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(1-pyrrolidinyl)-2-propanyl]octanamide
- External IDs
- GENZ 99067
- GENZ-99067
Pharmacology
- Indication
Eliglustat is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease in adult patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.6 CYP2D6 ultra-rapid metabolizers may not achieve adequate eliglustat concentrations to achieve a therapeutic effect. A specific dosage cannot be recommended for CYP2D6 indeterminate metabolizers.6
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- Contraindications & Blackbox Warnings
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Eliglustat is a specific inhibitor of glucosylceramide synthase (IC50 =10 ng/mL).6 In vitro studies suggest that eliglustat has minimal or no off-target activity against other glycosidases, such as α-glucosidase I and II, and lysosomal and non-lysosomal glucosylceramidases.3
At 8 times the recommended dose (800 mg) and a mean peak concentration of 237 ng/mL, eliglustat did not have a clinically significant effect on QTc prolongation. However, modelling of PK/PD data predicts that at a plasma concentration of 500 ng/mL, PR, QRS and QTcF intervals increase 22, 7, and 13 msec, respectively.6 Since high plasma concentrations of eliglustat may increase the risk of cardiac arrhythmias, there are warnings and precautions for patients taking CYP2D6 or CYP3A4 inhibitors, those with specific CYP2D6 metabolizer status and different degrees of hepatic impairment. Depending on each case, the use of this drug is contraindicated, to be avoided, or requires dosage adjustment.6
Patients with preexisting cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia), long QT syndrome, or those taking Class IA or Class II antiarrhythmic drugs are advised to avoid eliglustat.6
- Mechanism of action
Eliglustat is a glucosylceramide synthase inhibitor used for the treatment of type 1 Gaucher disease.6 Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide (also known as glucocerebroside) into glucose and ceramide. In patients with Gaucher disease, glucosylceramide is accumulated in the lysosomes of macrophages, leading to the formation of foam cells or Gaucher cells.6 Gaucher cells infiltrate the liver, spleen, bone marrow and other organs, leading to complications such as anemia, thrombocytopenia and hepatosplenomegaly.6,4
Eliglustat reduces the production of glucosylceramide by inhibiting glucosylceramide synthase, a rate-limiting enzyme in the production of glycosphingolipids.6,3 This lowers the amount of glucosylceramide that is available in lysosomes, and balances the deficiency of acid β-glucosidase.6,4
Target Actions Organism ACeramide glucosyltransferase inhibitorHumans - Absorption
Eliglustat administered in multiple doses of 84 mg twice daily had a Cmax of 12.1 to 25.0 ng/mL in CYP2D6 extensive metabolizers (EMs), 44.6 ng/mL in CYP2D6 intermediate metabolizers (IMs), and 113 to 137 ng/mL in CYP2D6 poor metabolizers (PMs).6 The median Tmax was 1.5-2 hr in CYP2D6 EMs, 2 hr in CYP2D6 IMs, and 3 hr in CYP2D6 PMs.6 The AUCtau was 76.3-143 ng∙hr/mL in CYP2D6 EMs, 306 ng∙hr/mL in CYP2D6 IMs, and 922-1057 ng∙hr/mL in CYP2D6 PMs.6 In CYP2D6 EMs, the pharmacokinetics of eliglustat is time-dependent, and for doses that range between 42 and 294 mg, exposure increases in a more than dose-proportional fashion. In CYP2D6 PMs, eliglustat pharmacokinetics is linear and time-independent. In a steady state, the systemic exposure of 84 mg eliglustat twice daily is 7- to 9-fold higher in CYP2D6 PMs compared to EMs.6
Following the oral administration of a single 84 mg dose of eliglustat, bioavailability in CYP2D6 EMs was lower than 5%.6 The low oral bioavailability of eliglustat suggests the role of transporters and/or an extensive first-pass metabolism.7 Eliglustat can be taken with or without food.6 In CYP2D6 EMs, severe renal impairment did not have an effect on eliglustat pharmacokinetics. The effect of renal impairment on eliglustat pharmacokinetics was not evaluated in CYP2D6 IMs, CYP2D6 PMs or CYP2D6 EMs with end-stage renal failure.6 Compared to CYP2D6 EMs with normal hepatic function, Cmax and AUC were 1.2-fold higher in CYP2D6 EMs with mild hepatic impairment, while Cmax and AUC were 2.8- and 5.2-fold higher, respectively, in CYP2D6 EMs with moderate hepatic impairment. The effect of mild and moderate hepatic impairment in CYP2D6 IMs and PMs, and the effect of severe hepatic impairment were not evaluated.6
- Volume of distribution
In CYP2D6 extensive metabolizers (EM), the volume of distribution of eliglustat administered IV was 835 L.6
- Protein binding
In plasma, the protein binding of eliglustat goes from 76% to 83%.6
- Metabolism
Eliglustat is mostly metabolized by CYP2D6, and to a lower extent, by CYP3A4.6 In patients that are CYP2D6 poor metabolizers (PMs), eliglustat is mainly metabolized by CYP3A4. The primary metabolic pathways of eliglustat involve the sequential oxidation of the octanoyl moiety and the 2,3-dihydro-1,4-benzodioxane moiety. The combination of these two pathways results in the production of several oxidative metabolites.8
After evaluating the potency of eliglustat metabolites, it was determined that none of them were active.8 Genz-399240 (M24) was identified as the major metabolite of eliglustat, while the rest of the metabolites contributed to less than 10% of total drug-related exposures.7 Genz-399240 (M24) did not show any major off-target effects; therefore, a transporter substrate specificity characterization was not performed.7
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- Route of elimination
Eliglustat is mainly excreted in urine (42%) and feces (51%) as metabolites after oral administration.6
- Half-life
Eliglustat has a terminal elimination half-life of 6.5 hours in CYP2D6 extensive metabolizers (EMs) and 8.9 h in CYP2D6 poor metabolizers (PMs).6
- Clearance
In healthy CYP2D6 extensive metabolizers (EMs) administered 42 mg of eliglustat IV (0.5 times the recommended oral dose), clearance was 88 L/h (80-105 L/h).6
- Adverse Effects
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates.Improve decision support & research outcomes with our structured adverse effects data.- Toxicity
Eliglustat overdose may manifest as dizziness marked by disequilibrium, hypotension, bradycardia, nausea, and vomiting. These symptoms were detected in a healthy subject taking 21-times the dose recommended to type 1 Gaucher disease patients.6 Eliglustat has no known antidote. In case of acute overdose, the patient should be carefully observed and given symptomatic and supportive treatment.6 Due to the large volume of distribution of eliglustat, hemodialysis is not likely to be beneficial.6
Acute dose toxicity studies were performed in rats and dogs. In rats, the maximum tolerated dose was 200 mg/kg, and in non-fasted dogs, the maximum tolerated dose was 25 mg/kg.7 Some of the adverse effects detected in these toxicity studies manifested on the GI tract, hematology parameters related to hemoglobin and coagulation process, reproductive organs, thymus and other lymphoid organs. Adverse effects in the kidney and liver were only detected in rats.7
Carcinogenic studies were performed in both Sprague-Dawley rats and CD-1 mice. In doses up to 50 mg/kg/day in female Sprague-Dawley rats and 75 mg/kg/day in male Sprague-Dawley rats and CD-1 mice, eliglustat did not induce neoplasms.6 Eliglustat was negative in the following mutagenesis tests: Ames test, chromosome aberration test in human peripheral blood lymphocytes, mouse lymphoma gene mutation assay and in vivo oral mouse micronucleus test.6
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
Interacting Gene/Enzyme Allele name Genotype(s) Defining Change(s) Type(s) Description Details Cytochrome P450 2D6 CYP2D6*3 Not Available C allele Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*4 Not Available C allele Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*5 Not Available Whole-gene deletion Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*6 Not Available 1707delT Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*7 Not Available 2935A>C Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*8 Not Available 1758G>T Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*11 Not Available 883G>C Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*12 Not Available 124G>A Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*13 Not Available CYP2D7/2D6 hybrid gene structure Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*14A Not Available 1758G>A Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*15 Not Available 137insT, 137_138insT Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*19 Not Available 2539_2542delAACT Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*20 Not Available 1973_1974insG Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*21 Not Available 2573insC Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*31 Not Available -1770G>A / -1584C>G … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*36 Not Available 100C>T / -1426C>T … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*38 Not Available 2587_2590delGACT Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*40 Not Available 1863_1864ins(TTT CGC CCC)2 Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*42 Not Available 3259_3260insGT Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*44 Not Available 2950G>C Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*47 Not Available 100C>T / -1426C>T … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*51 Not Available -1584C>G / -1235A>G … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*56 Not Available 3201C>T Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*57 Not Available 100C>T / 310G>T … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*62 Not Available 4044C>T Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*68A Not Available -1426C>T / -1235A>G … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*68B Not Available Similar but not identical switch region compared to CYP2D6*68A. Found in tandem arrangement with CYP2D6*4. Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*69 Not Available 2988G>A / -1426C>T … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*92 Not Available 1995delC Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*100 Not Available -1426C>T / -1235A>G … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*101 Not Available -1426C>T / -1235A>G … show all Effect Inferred Poor drug metabolizer, lower dose requirement. Details Cytochrome P450 2D6 CYP2D6*1XN Not Available Normal allele duplicated. Effect Inferred Reduced efficacy. Details Cytochrome P450 2D6 CYP2D6*2XN Not Available 2850C>T / 4180G>C … show all Effect Inferred Reduced efficacy. Details
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Drug Interaction Integrate drug-drug
interactions in your softwareAbametapir The serum concentration of Eliglustat can be increased when it is combined with Abametapir. Abatacept The metabolism of Eliglustat can be increased when combined with Abatacept. Abemaciclib The serum concentration of Abemaciclib can be increased when it is combined with Eliglustat. Abiraterone The metabolism of Eliglustat can be decreased when combined with Abiraterone. Acalabrutinib The metabolism of Eliglustat can be decreased when combined with Acalabrutinib. Acebutolol The metabolism of Acebutolol can be decreased when combined with Eliglustat. Acetaminophen The metabolism of Eliglustat can be decreased when combined with Acetaminophen. Acetazolamide The metabolism of Eliglustat can be decreased when combined with Acetazolamide. Acrivastine The risk or severity of QTc prolongation can be increased when Acrivastine is combined with Eliglustat. Adalimumab The metabolism of Eliglustat can be increased when combined with Adalimumab. 
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- Avoid grapefruit products. Grapefruit products are strong CYP3A inhibitors and may increase the concentration of eliglustat.
- Avoid St. John's Wort. This herb induces the CYP3A metabolism of eliglustat and may reduce its serum concentration.
- Take with or without food. Administration of eliglustat with a high-fat meal resulted in a 15% decrease in Cmax (not clinically significant) and no change in AUC.
Products
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Ingredient UNII CAS InChI Key Eliglustat tartrate N0493335P3 928659-70-5 KUBARPMUNHKBIQ-VTHUDJRQSA-N - International/Other Brands
- Cerdelga (Genzyme Corporation)
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Cerdelga Capsule 84 mg Oral Genzyme Europe Bv 2016-09-08 Not applicable EU 
Cerdelga Capsule 84 mg Oral Genzyme Europe Bv 2016-09-08 Not applicable EU Cerdelga Capsule 84 mg Oral Sanofi Genzyme, a Division of Sanofi Aventis Canada Inc 2017-07-26 Not applicable Canada 
Cerdelga Capsule 84 mg Oral Genzyme Europe Bv 2016-09-08 Not applicable EU Cerdelga Capsule 84 mg/1 Oral Genzyme Corporation 2014-09-03 Not applicable US 
Categories
- ATC Codes
- A16AX10 — Eliglustat
- Drug Categories
- Alimentary Tract and Metabolism
- Cytochrome P-450 CYP2D6 Inhibitors
- Cytochrome P-450 CYP2D6 Inhibitors (strength unknown)
- Cytochrome P-450 CYP2D6 Substrates
- Cytochrome P-450 CYP3A Substrates
- Cytochrome P-450 CYP3A4 Substrates
- Cytochrome P-450 Enzyme Inhibitors
- Cytochrome P-450 Substrates
- Enzyme Inhibitors
- Gaucher Disease
- Glucosylceramide Synthase Inhibitor
- Glucosylceramide Synthase Inhibitors
- Highest Risk QTc-Prolonging Agents
- Other Miscellaneous Therapeutic Agents
- P-glycoprotein inhibitors
- P-glycoprotein substrates
- QTc Prolonging Agents
- Various Alimentary Tract and Metabolism Products
- Chemical TaxonomyProvided by Classyfire
- Description
- This compound belongs to the class of organic compounds known as benzo-1,4-dioxanes. These are heterocyclic compounds containing a benzene ring fused to a 1,4-dioxane ring.
- Kingdom
- Organic compounds
- Super Class
- Organoheterocyclic compounds
- Class
- Benzodioxanes
- Sub Class
- Benzo-1,4-dioxanes
- Direct Parent
- Benzo-1,4-dioxanes
- Alternative Parents
- Alkyl aryl ethers / Aralkylamines / Para dioxins / N-alkylpyrrolidines / N-acyl amines / Benzenoids / 1,3-aminoalcohols / Trialkylamines / Secondary carboxylic acid amides / Secondary alcohols show 8 more
- Substituents
- 1,3-aminoalcohol / Alcohol / Alkyl aryl ether / Amine / Amino acid or derivatives / Aralkylamine / Aromatic alcohol / Aromatic heteropolycyclic compound / Azacycle / Benzenoid show 23 more
- Molecular Framework
- Aromatic heteropolycyclic compounds
- External Descriptors
- secondary alcohol, carboxamide, N-alkylpyrrolidine, benzodioxine (CHEBI:82752)
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- DR40J4WA67
- CAS number
- 491833-29-5
- InChI Key
- FJZZPCZKBUKGGU-AUSIDOKSSA-N
- InChI
- InChI=1S/C23H36N2O4/c1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20/h10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26)/t19-,23-/m1/s1
- IUPAC Name
- N-[(1R,2R)-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl]octanamide
- SMILES
- CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)C1=CC=C2OCCOC2=C1
References
- Synthesis Reference
Bradford, HH., et al. (2005). Synthesis of UDP-Glucose: N-Acylsphingosine glucosyltransferase inhibitors (U.S. Patent No. 6,855,830 B2). U.S. Patent and Trademark Office. https://patentimages.storage.googleapis.com/ef/ef/57/109e82e31de7f9/US6855830.pdf
- General References
- Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
- McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J: A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. Epub 2007 May 16. [Article]
- Scott LJ: Eliglustat: A Review in Gaucher Disease Type 1. Drugs. 2015 Sep;75(14):1669-78. doi: 10.1007/s40265-015-0468-9. [Article]
- Dandana A, Ben Khelifa S, Chahed H, Miled A, Ferchichi S: Gaucher Disease: Clinical, Biological and Therapeutic Aspects. Pathobiology. 2016;83(1):13-23. doi: 10.1159/000440865. Epub 2015 Nov 21. [Article]
- Nalysnyk L, Sugarman R, Cele C, Uyei J, Ward A: Budget Impact Analysis of Eliglustat for the Treatment of Gaucher Disease Type 1 in the United States. J Manag Care Spec Pharm. 2018 Oct;24(10):1002-1008. doi: 10.18553/jmcp.2018.24.10.1002. [Article]
- FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
- EMA Assessment Report: Cerdelga (eliglustat) oral capsules [Link]
- EMA Summary of Product Characteristics: Cerdelga (eliglustat) oral capsules [Link]
- Health Canada Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
- External Links
- KEGG Drug
- D09893
- PubChem Compound
- 23652731
- PubChem Substance
- 310264987
- ChemSpider
- 28475348
- 1547220
- ChEBI
- 82752
- ChEMBL
- CHEMBL2110588
- ZINC
- ZINC000072267023
- PharmGKB
- PA166123486
- Wikipedia
- Eliglustat
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 3 Active Not Recruiting Treatment Gaucher Disease, Type 1 / Gaucher Disease, Type III 1 3 Completed Treatment Gaucher Disease, Type 1 2 3 Completed Treatment Gaucher's Disease 2 2 Completed Treatment Cerebroside Lipidosis Syndrome / Gaucher Disease, Non-Neuronopathic Form / Gaucher Disease, Type 1 / Glucocerebrosidase Deficiency Disease / Glucosylceramide Beta-Glucosidase Deficiency Disease 1 1 Completed Not Available Healthy Subjects (HS) 1 1 Completed Basic Science Healthy Subjects (HS) 1 1 Completed Treatment Gaucher's Disease 3 1 Completed Treatment Healthy Subjects (HS) 1 1 Recruiting Treatment Malignancies, Hematologic / Solid Tumors 1 Not Available Completed Treatment Gaucher Disease, Type III 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Capsule Oral 84 mg/1 Capsule Oral Capsule, coated Oral 84 mg Capsule Oral 84 mg - Prices
- Not Available
- Patents
Patent Number Pediatric Extension Approved Expires (estimated) Region US6916802 No 2005-07-12 2022-04-29 US US7615573 No 2009-11-10 2022-04-29 US US7196205 No 2007-03-27 2022-04-29 US US7253185 No 2007-08-07 2022-04-29 US US10888547 No 2021-01-12 2031-01-31 US US10888544 No 2021-01-12 2038-12-13 US
Properties
- State
- Solid
- Experimental Properties
- Not Available
- Predicted Properties
Property Value Source Water Solubility 0.113 mg/mL ALOGPS logP 3.44 ALOGPS logP 3.21 Chemaxon logS -3.6 ALOGPS pKa (Strongest Acidic) 13.51 Chemaxon pKa (Strongest Basic) 8.17 Chemaxon Physiological Charge 1 Chemaxon Hydrogen Acceptor Count 5 Chemaxon Hydrogen Donor Count 2 Chemaxon Polar Surface Area 71.03 Å2 Chemaxon Rotatable Bond Count 11 Chemaxon Refractivity 113.76 m3·mol-1 Chemaxon Polarizability 46.82 Å3 Chemaxon Number of Rings 3 Chemaxon Bioavailability 1 Chemaxon Rule of Five Yes Chemaxon Ghose Filter Yes Chemaxon Veber's Rule No Chemaxon MDDR-like Rule Yes Chemaxon - Predicted ADMET Features
- Not Available
Spectra
- Mass Spec (NIST)
- Not Available
- Spectra
Spectrum Spectrum Type Splash Key Predicted MS/MS Spectrum - 10V, Positive (Annotated) Predicted LC-MS/MS Not Available Predicted MS/MS Spectrum - 20V, Positive (Annotated) Predicted LC-MS/MS Not Available Predicted MS/MS Spectrum - 40V, Positive (Annotated) Predicted LC-MS/MS Not Available Predicted MS/MS Spectrum - 10V, Negative (Annotated) Predicted LC-MS/MS Not Available Predicted MS/MS Spectrum - 20V, Negative (Annotated) Predicted LC-MS/MS Not Available Predicted MS/MS Spectrum - 40V, Negative (Annotated) Predicted LC-MS/MS Not Available
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Yes
- Actions
- Inhibitor
- General Function
- Ceramide glucosyltransferase activity
- Specific Function
- Catalyzes the first glycosylation step in glycosphingolipid biosynthesis, the transfer of glucose to ceramide. May also serve as a "flippase".
- Gene Name
- UGCG
- Uniprot ID
- Q16739
- Uniprot Name
- Ceramide glucosyltransferase
- Molecular Weight
- 44853.255 Da
References
Enzymes
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- No
- Actions
- SubstrateInhibitor
- General Function
- Steroid hydroxylase activity
- Specific Function
- Responsible for the metabolism of many drugs and environmental chemicals that it oxidizes. It is involved in the metabolism of drugs such as antiarrhythmics, adrenoceptor antagonists, and tricyclic...
- Gene Name
- CYP2D6
- Uniprot ID
- P10635
- Uniprot Name
- Cytochrome P450 2D6
- Molecular Weight
- 55768.94 Da
References
- Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
- FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
- Flockhart Table of Drug Interactions [Link]
- FDA Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers [Link]
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- No
- Actions
- Substrate
- General Function
- Vitamin d3 25-hydroxylase activity
- Specific Function
- Cytochromes P450 are a group of heme-thiolate monooxygenases. In liver microsomes, this enzyme is involved in an NADPH-dependent electron transport pathway. It performs a variety of oxidation react...
- Gene Name
- CYP3A4
- Uniprot ID
- P08684
- Uniprot Name
- Cytochrome P450 3A4
- Molecular Weight
- 57342.67 Da
References
- Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
- FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
- Flockhart Table of Drug Interactions [Link]
- FDA Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers [Link]
Transporters
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- No
- Actions
- SubstrateInhibitor
- General Function
- Xenobiotic-transporting atpase activity
- Specific Function
- Energy-dependent efflux pump responsible for decreased drug accumulation in multidrug-resistant cells.
- Gene Name
- ABCB1
- Uniprot ID
- P08183
- Uniprot Name
- Multidrug resistance protein 1
- Molecular Weight
- 141477.255 Da
References
Drug created at April 02, 2015 16:54 / Updated at November 26, 2022 07:39