eliglustat 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | 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

Approvals:

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

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 136.09 37.68 27 524 7585 63480886
Product dose omission issue 70.56 37.68 36 515 234277 63254194

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 197.06 53.03 31 289 2243 34954368
Product dose omission issue 85.64 53.03 34 286 119677 34836934

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 220.95 40.43 41 686 7747 79735914
Product dose omission issue 115.15 40.43 52 675 247485 79496176
Weight decreased 49.85 40.43 34 693 355164 79388497

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:50382 glucosylceramide synthase inhibitors

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 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
CHEMBL4297066 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

Pharmaceutical products:

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