migalastat Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definitionDrug idCAS RN
enzyme inhibitors 5110 108147-54-2

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • migalastat HCl
  • GR181413A
  • 1-Deoxygalactonojirimycin hydrochloride
  • 1-Deoxygalactonojirimycin
  • migalastat
  • migalastat hydrochloride
  • galafold
Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Migalastat binding stabilizes these mutant forms of alpha-Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes where dissociation of migalastat restores alpha-Gal A activity, leading to the catabolism of GL-3 and related substrates.
  • Molecular weight: 163.17
  • Formula: C6H13NO4
  • CLOGP: -1.42
  • LIPINSKI: 0
  • HAC: 5
  • HDO: 5
  • TPSA: 92.95
  • ALOGS: 0.50
  • ROTB: 1

  • Status: ONP

  • Legend:
    OFP - off patent
    OFM - off market
    ONP - on patent

Drug dosage:

DoseUnitRoute
61.50 mg O

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
Aug. 8, 2018 FDA AMICUS THERAPS US
March 23, 2018 PMDA Amicus Therapeutics Inc
May 26, 2016 EMA Amicus Therapeutics UK Ltd

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

Pharmacologic Action:

SourceCodeDescription
ATC A16AX14 ALIMENTARY TRACT AND METABOLISM
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Various alimentary tract and metabolism products

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Fabry's disease indication 16652001 DOID:14499

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 12.16 acidic
pKa2 8.5 Basic

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
EQ 123MG BASE GALAFOLD AMICUS THERAPS US N208623 Aug. 10, 2018 RX CAPSULE ORAL Aug. 10, 2023 NEW CHEMICAL ENTITY
EQ 123MG BASE GALAFOLD AMICUS THERAPS US N208623 Aug. 10, 2018 RX CAPSULE ORAL Aug. 10, 2025 INDICATED FOR THE TREATMENT OF ADULTS WITH A CONFIRMED DIAGNOSIS OF FABRY DISEASE AND AN AMENULLBLE GALACTOSIDASE ALPHA GENE (GLA) VARIANT BASED ON IN VITRO ASSAY DATA

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Alpha-galactosidase A Enzyme PHARMACOLOGICAL CHAPERONE IC50 7.15 CHEMBL DRUG LABEL
Beta-galactosidase Enzyme IC50 4.05 CHEMBL
Lactase-phlorizin hydrolase Enzyme IC50 4.30 CHEMBL
Glycogen debranching enzyme Enzyme IC50 5 CHEMBL
Sucrase-isomaltase, intestinal Enzyme IC50 6.59 CHEMBL
Non-lysosomal glucosylceramidase Enzyme IC50 4 CHEMBL
Lysosomal alpha-glucosidase Enzyme IC50 5.22 CHEMBL
Maltase-glucoamylase, intestinal Enzyme IC50 5.82 CHEMBL
Beta-galactosidase Unclassified IC50 4.62 CHEMBL
Lactase-phlorizin hydrolase Unclassified IC50 4.14 CHEMBL
Alpha-galactosidase Unclassified IC50 8.52 CHEMBL
Beta-galactosidase Unclassified Ki 4.90 CHEMBL
Alpha-galactosidase C Unclassified IC50 5.74 CHEMBL
Beta-galactosidase Unclassified Ki 4.89 CHEMBL

External reference:

IDSource
75172-81-5 SECONDARY_CAS_RN
D10359 KEGG_DRUG
4038521 VANDF
C0754022 UMLSCUI
CHEBI:135923 CHEBI
DGJ PDB_CHEM_ID
176077 PUBCHEM_CID
CHEMBL110458 ChEMBL_ID
CHEMBL2107355 ChEMBL_ID
DB05018 DRUGBANK_ID
C090092 MESH_SUPPLEMENTAL_RECORD_UI
10200 IUPHAR_LIGAND_ID
8700 INN_ID
C4XNY919FW UNII
2054252 RXNORM
261328 MMSL
33035 MMSL
d08640 MMSL
017003 NDDF
017004 NDDF
765340000 SNOMEDCT_US
765341001 SNOMEDCT_US
773543004 SNOMEDCT_US

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Galafold HUMAN PRESCRIPTION DRUG LABEL 1 71904-100 CAPSULE 123 mg ORAL NDA 26 sections