eliglustat 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definitionDrug idCAS RN
enzyme inhibitors 4834 491833-29-5

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • cerdelga
  • eliglustat
  • Genz-99067
  • eliglustat tartrate
  • Genz-112638
Eliglustat tartrate is a small molecule inhibitor of glucosylceramide synthase that resembles the ceramide substrate for the enzyme. Gaucher disease is caused by a deficiency of the lysosomal enzyme acid beta-glucosidase. Acid beta-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells".
  • Molecular weight: 404.55
  • Formula: C23H36N2O4
  • CLOGP: 4.83
  • LIPINSKI: 0
  • HAC: 6
  • HDO: 2
  • TPSA: 71.03
  • ALOGS: -3.55
  • ROTB: 11
  • Status: OFP
    • Legend:
    OFP - off patent
    OFM - off market
    ONP - on patent

Drug dosage:

DoseUnitRoute
0.17 g O

ADMET properties:

PropertyValueReference
CL (Clearance) 20.95 mL/min/kg Lombardo F, Berellini G, Obach RS
fu (Fraction unbound in plasma) 0.21 % Lombardo F, Berellini G, Obach RS
t_half (Half-life) 7.70 hours Lombardo F, Berellini G, Obach RS
BA (Bioavailability) 83 %

Predicted pharmacokinetic/toxicological properties (PK-AZ):

PropertyDescriptionValueUnitConfidenceSimilarity
azlogd74 LogD at pH 7.4 2.720 High 1
caco2-efflux Caco-2 efflux ratio (BA/AB) 8.110 High 1
caco2-intrinsic-papp Caco-2 intrinsic apparent permeability 13.677 10^-6 cm/s High 1
dh-clint Dog hepatocyte intrinsic clearance 14.028 uL/min/10^6 cells High 1
dppb Dog plasma protein binding (% unbound) 6.530 % High 1
fcs-ppb Fetal calf serum protein binding (% unbound) 20.560 % High 1
herg hERG pIC50 5.366 pIC50 High 1
hh-clint Human hepatocyte intrinsic clearance 18.621 uL/min/10^6 cells High 1
hlm-clint Human liver microsomal intrinsic clearance 116.413 uL/min/mg protein High 1
hppb Human plasma protein binding (% unbound) 6.960 % High 1
kinase-safety-class ACVR2B,AXL,BRAF,BTK,CAMK2B,CAMK2D,CDK4,CDK5,CDK9,EIF2AK3,EPHB4,ERBB2,GRK2,ITK,MAPK10,MAPK7,MET,NTRK2,PDK2,PDK4,PIK3CA,PIK3CB,PRKDC,TEK,TGFBR1 25
kinase-selectivity-class AXL,PDK4 2
mdck-mdr1-efflux MDCK-MDR1 efflux ratio 8.204 High 1
mh-clint Mouse hepatocyte intrinsic clearance 96.605 High 1
mppb Mouse plasma protein binding (% unbound) 10.170 High 1
nih-mdck-mdr1-efflux NIH MDCK-MDR1 efflux ratio 33.806 High 1
pfas-class PFAS structural alert (1 = True, 0 = False) 0
pppb Pig plasma protein binding (% unbound) 20.340 % High 1
rh-clint Rat hepatocyte intrinsic clearance 175.388 uL/min/10^6 cells High 1
rh-fuinc Rat hepatocyte fraction unbound in incubation 35.730 % High 1
rppb Rat plasma protein binding (% unbound) 8.960 % High 1
solubility-dd Solubility at pH 7.4 in DMSO 796.159 uM High 1

Approvals:

DateAgencyCompanyOrphan
Aug. 19, 2014 FDA GENZYME CORP
Nov. 20, 2014 EMA Genzyme Europe BV

FDA Adverse Event Reporting System (Female)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Product dose omission in error 245.51 38.39 55 1007 20448 90606937
Weight increased 120.42 38.39 69 993 417858 90209527
Weight decreased 99.35 38.39 59 1003 379851 90247534

FDA Adverse Event Reporting System (Male)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Product dose omission in error 282.13 43.49 55 585 8299 42612396
Weight increased 202.87 43.49 71 569 107912 42512783
Weight decreased 105.13 43.49 54 586 207894 42412801

FDA Adverse Event Reporting System (Geriatric)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Product dose omission in error 408.04 37.10 87 1344 23425 110564443
Weight increased 227.22 37.10 109 1322 419535 110168333
Weight decreased 164.23 37.10 91 1340 467966 110119902
Product dose omission issue 52.84 37.10 39 1392 321743 110266125
Intentional dose omission 50.27 37.10 16 1415 20064 110567804

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC A16AX10 ALIMENTARY TRACT AND METABOLISM
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Various alimentary tract and metabolism products
FDA MoA N0000020019 Glucosylceramide Synthase Inhibitors
MeSH PA D004791 Enzyme Inhibitors
FDA EPC N0000175783 Glucosylceramide Synthase Inhibitor
FDA MoA N0000182137 Cytochrome P450 2D6 Inhibitors
FDA MoA N0000185503 P-Glycoprotein Inhibitors
CHEBI has role CHEBI:35610 antineoplastic agent
CHEBI has role CHEBI:50382 EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor

Related Drugs by ATC class:

A16AX Various alimentary tract and metabolism products 23 drugs
anethole trithionedoxecitinedoxribtiminefosdenopteringivosiranglycerol phenylbutyratelonafarniblumasiranmigalastatmiglustatnedosirannitisinone show all 23 →

Drug Use | Suggest Off label Use Form| |View source of the data|

DiseaseRelationSNOMED_IDDOID
Glucosylceramide beta-glucosidase deficiency indication 190794006 DOID:1926




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 13.37 acidic
pKa2 8.14 Basic

Orange Book patent data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 10888547 Jan. 31, 2031 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 EXTENSIVE OR INTERMEDIATE METABOLIZERS WITH 84 MG TWICE PER DAY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE TWICE PER DAY)
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 10888547 Jan. 31, 2031 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 POOR METABOLIZERS WITH 84 MG ONCE DAILY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE)
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 12465586 June 5, 2032 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 EXTENSIVE OR INTERMEDIATE METABOLIZERS WITH 84 MG TWICE PER DAY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE TWICE PER DAY)
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 12465586 June 5, 2032 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 POOR METABOLIZERS WITH 84 MG ONCE DAILY OF ELIGLUSTAT (EQUIVALENT TO 100 MG OF ELIGLUSTAT TARTRATE)
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 10888544 Dec. 13, 2038 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 EXTENSIVE METABOLIZERS WITH MILD HEPATIC IMPAIRMENT AND ARE CONCURRENTLY TAKING A STRONG OR MODERATE CYP3A INHIBITOR
EQ 84MG BASE CERDELGA GENZYME CORP N205494 Aug. 19, 2014 RX CAPSULE ORAL 10888544 Dec. 13, 2038 LONG-TERM TREATMENT OF ADULTS WITH GAUCHER DISEASE TYPE 1 WHO ARE CYP2D6 EXTENSIVE METABOLIZERS WITH MODERATE TO SEVERE RENAL IMPAIRMENT

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])		Mechanism
action		Bioact sourceMoA source
Ceramide glucosyltransferase Enzyme INHIBITOR IC50 6.83 SCIENTIFIC LITERATURE SCIENTIFIC LITERATURE

External reference:

IDSource
DR40J4WA67 UNII
D09893 KEGG_DRUG
928659-70-5 SECONDARY_CAS_RN
4033647 VANDF
C3272698 UMLSCUI
CHEBI:82752 CHEBI
CHEMBL2110588 ChEMBL_ID
23652731 PUBCHEM_CID
DB09039 DRUGBANK_ID
CHEMBL5723563 ChEMBL_ID
9325 INN_ID
C522917 MESH_SUPPLEMENTAL_RECORD_UI
7536 IUPHAR_LIGAND_ID
1547219 RXNORM
228048 MMSL
30495 MMSL
33941 MMSL
d08283 MMSL
015653 NDDF
015654 NDDF
714006001 SNOMEDCT_US
714011004 SNOMEDCT_US
863995003 SNOMEDCT_US
DR40J4WA67 INXIGHT DRUGS

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Cerdelga HUMAN PRESCRIPTION DRUG LABEL 1 58468-0220 CAPSULE 84 mg ORAL NDA 30 sections