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
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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Associated Conditions
Contraindications & Blackbox Warnings
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Pharmacodynamics

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

TargetActionsOrganism
ACeramide glucosyltransferase
inhibitor
Humans
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
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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/EnzymeAllele nameGenotype(s)Defining Change(s)Type(s)DescriptionDetails
Cytochrome P450 2D6CYP2D6*3Not AvailableC alleleEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*4Not AvailableC alleleEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*5Not AvailableWhole-gene deletionEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*6Not Available1707delTEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*7Not Available2935A>CEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*8Not Available1758G>TEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*11Not Available883G>CEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*12Not Available124G>AEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*13Not AvailableCYP2D7/2D6 hybrid gene structureEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*14ANot Available1758G>AEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*15Not Available137insT, 137_138insTEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*19Not Available2539_2542delAACTEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*20Not Available1973_1974insGEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*21Not Available2573insCEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*31Not Available-1770G>A / -1584C>G  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*36Not Available100C>T / -1426C>T  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*38Not Available2587_2590delGACTEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*40Not Available1863_1864ins(TTT CGC CCC)2Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*42Not Available3259_3260insGTEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*44Not Available2950G>CEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*47Not Available100C>T / -1426C>T  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*51Not Available-1584C>G / -1235A>G  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*56Not Available3201C>TEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*57Not Available100C>T / 310G>T  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*62Not Available4044C>TEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*68ANot Available-1426C>T / -1235A>G  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*68BNot AvailableSimilar but not identical switch region compared to CYP2D6*68A. Found in tandem arrangement with CYP2D6*4.Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*69Not Available2988G>A / -1426C>T  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*92Not Available1995delCEffect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*100Not Available-1426C>T / -1235A>G  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*101Not Available-1426C>T / -1235A>G  … show all Effect InferredPoor drug metabolizer, lower dose requirement.Details
Cytochrome P450 2D6CYP2D6*1XNNot AvailableNormal allele duplicated.Effect InferredReduced efficacy.Details
Cytochrome P450 2D6CYP2D6*2XNNot Available2850C>T / 4180G>C  … show all Effect InferredReduced 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.
DrugInteraction
AbametapirThe serum concentration of Eliglustat can be increased when it is combined with Abametapir.
AbataceptThe metabolism of Eliglustat can be increased when combined with Abatacept.
AbemaciclibThe serum concentration of Abemaciclib can be increased when it is combined with Eliglustat.
AbirateroneThe metabolism of Eliglustat can be decreased when combined with Abiraterone.
AcalabrutinibThe metabolism of Eliglustat can be decreased when combined with Acalabrutinib.
AcebutololThe metabolism of Acebutolol can be decreased when combined with Eliglustat.
AcetaminophenThe metabolism of Eliglustat can be decreased when combined with Acetaminophen.
AcetazolamideThe metabolism of Eliglustat can be decreased when combined with Acetazolamide.
AcrivastineThe risk or severity of QTc prolongation can be increased when Acrivastine is combined with Eliglustat.
AdalimumabThe metabolism of Eliglustat can be increased when combined with Adalimumab.
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Food Interactions
  • 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.

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Product Ingredients
IngredientUNIICASInChI Key
Eliglustat tartrateN0493335P3928659-70-5KUBARPMUNHKBIQ-VTHUDJRQSA-N
International/Other Brands
Cerdelga (Genzyme Corporation)
Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
CerdelgaCapsule84 mgOralGenzyme Europe Bv2016-09-08Not applicableEU flag
CerdelgaCapsule84 mgOralGenzyme Europe Bv2016-09-08Not applicableEU flag
CerdelgaCapsule84 mgOralSanofi Genzyme, a Division of Sanofi Aventis Canada Inc2017-07-26Not applicableCanada flag
CerdelgaCapsule84 mgOralGenzyme Europe Bv2016-09-08Not applicableEU flag
CerdelgaCapsule84 mg/1OralGenzyme Corporation2014-09-03Not applicableUS flag

Categories

ATC Codes
A16AX10 — Eliglustat
Drug Categories
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
  1. Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
  2. 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]
  3. 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]
  4. 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]
  5. 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]
  6. FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
  7. EMA Assessment Report: Cerdelga (eliglustat) oral capsules [Link]
  8. EMA Summary of Product Characteristics: Cerdelga (eliglustat) oral capsules [Link]
  9. Health Canada Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
KEGG Drug
D09893
PubChem Compound
23652731
PubChem Substance
310264987
ChemSpider
28475348
RxNav
1547220
ChEBI
82752
ChEMBL
CHEMBL2110588
ZINC
ZINC000072267023
PharmGKB
PA166123486
Wikipedia
Eliglustat

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
3Active Not RecruitingTreatmentGaucher Disease, Type 1 / Gaucher Disease, Type III1
3CompletedTreatmentGaucher Disease, Type 12
3CompletedTreatmentGaucher's Disease2
2CompletedTreatmentCerebroside Lipidosis Syndrome / Gaucher Disease, Non-Neuronopathic Form / Gaucher Disease, Type 1 / Glucocerebrosidase Deficiency Disease / Glucosylceramide Beta-Glucosidase Deficiency Disease1
1CompletedNot AvailableHealthy Subjects (HS)1
1CompletedBasic ScienceHealthy Subjects (HS)1
1CompletedTreatmentGaucher's Disease3
1CompletedTreatmentHealthy Subjects (HS)1
1RecruitingTreatmentMalignancies, Hematologic / Solid Tumors1
Not AvailableCompletedTreatmentGaucher Disease, Type III1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
CapsuleOral84 mg/1
CapsuleOral
Capsule, coatedOral84 mg
CapsuleOral84 mg
Prices
Not Available
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)Region
US6916802No2005-07-122022-04-29US flag
US7615573No2009-11-102022-04-29US flag
US7196205No2007-03-272022-04-29US flag
US7253185No2007-08-072022-04-29US flag
US10888547No2021-01-122031-01-31US flag
US10888544No2021-01-122038-12-13US flag

Properties

State
Solid
Experimental Properties
Not Available
Predicted Properties
PropertyValueSource
Water Solubility0.113 mg/mLALOGPS
logP3.44ALOGPS
logP3.21Chemaxon
logS-3.6ALOGPS
pKa (Strongest Acidic)13.51Chemaxon
pKa (Strongest Basic)8.17Chemaxon
Physiological Charge1Chemaxon
Hydrogen Acceptor Count5Chemaxon
Hydrogen Donor Count2Chemaxon
Polar Surface Area71.03 Å2Chemaxon
Rotatable Bond Count11Chemaxon
Refractivity113.76 m3·mol-1Chemaxon
Polarizability46.82 Å3Chemaxon
Number of Rings3Chemaxon
Bioavailability1Chemaxon
Rule of FiveYesChemaxon
Ghose FilterYesChemaxon
Veber's RuleNoChemaxon
MDDR-like RuleYesChemaxon
Predicted ADMET Features
Not Available

Spectra

Mass Spec (NIST)
Not Available
Spectra
SpectrumSpectrum TypeSplash Key
Predicted MS/MS Spectrum - 10V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 10V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Negative (Annotated)Predicted LC-MS/MSNot Available

Targets

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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
  1. Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
  2. FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]

Enzymes

Kind
Protein
Organism
Humans
Pharmacological action
No
Actions
Substrate
Inhibitor
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
  1. Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
  2. FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
  3. Flockhart Table of Drug Interactions [Link]
  4. 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
  1. Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
  2. FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]
  3. Flockhart Table of Drug Interactions [Link]
  4. FDA Drug Development and Drug Interactions: Table of Substrates, Inhibitors and Inducers [Link]

Transporters

Kind
Protein
Organism
Humans
Pharmacological action
No
Actions
Substrate
Inhibitor
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
  1. Poole RM: Eliglustat: first global approval. Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3. [Article]
  2. FDA Approved Drug Products: Cerdelga (eliglustat) oral capsules [Link]

Drug created at April 02, 2015 16:54 / Updated at November 26, 2022 07:39