migalastat 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | 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: -0.40
  • 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:

PropertyValueReference
S (Water solubility) 200 mg/mL Bocci G, Oprea TI, Benet LZ
BDDCS (Biopharmaceutical Drug Disposition Classification System) 3 Bocci G, Oprea TI, Benet LZ
CL (Clearance) 2.22 mL/min/kg Kawashima H, Watanabe R, Esaki T, Kuroda M, Nagao C, Natsume-Kitatani Y, Ohashi R, Komura H, Mizuguchi K
BA (Bioavailability) 0.75 % Kawashima H, Watanabe R, Esaki T, Kuroda M, Nagao C, Natsume-Kitatani Y, Ohashi R, Komura H, Mizuguchi K

Predicted pharmacokinetic/toxicological properties (PK-AZ):

PropertyDescriptionValueUnitConfidenceSimilarity
azlogd74 LogD at pH 7.4 -0.938 High 0.86
caco2-efflux Caco-2 efflux ratio (BA/AB) 0.782 High 0.86
caco2-intrinsic-papp Caco-2 intrinsic apparent permeability 0.547 10^-6 cm/s High 0.86
dh-clint Dog hepatocyte intrinsic clearance 0.646 uL/min/10^6 cells High 0.86
dppb Dog plasma protein binding (% unbound) 73.630 % High 0.86
fcs-ppb Fetal calf serum protein binding (% unbound) 77.130 % High 0.86
herg hERG pIC50 4.562 pIC50 High 0.86
hh-clint Human hepatocyte intrinsic clearance 1.026 uL/min/10^6 cells High 0.86
hlm-clint Human liver microsomal intrinsic clearance 4.178 uL/min/mg protein High 0.86
hppb Human plasma protein binding (% unbound) 66.770 % High 0.86
kinase-safety-class ACVR2A,ACVR2B,AXL,BRAF,CAMK2D,CDK4,CDK5,CDK9,DDR1,DYRK1B,EIF2AK3,ERBB2,GRK2,ITK,MAP2K6,MAP3K21,MAPK10,MAPK7,NTRK1,NTRK2,PDK2,PDK4,PRKDC,SIK2,STK33,TEK,TGFBR1,ZAP70 28
kinase-selectivity-class ACVRL1,AXL,BMX,CDK1,CDK16,CDK2,CDK3,CDK4,CDK6,CDK7,CDK8,CDK9,CHUK,DYRK1A,IGF1R,INSR,IRAK1,IRAK3,IRAK4,MAP2K6,MAP3K14,MAP3K21,PDK1,PIK3CB,PIK3CD,TGFBR1,ZAP70 27
mdck-mdr1-efflux MDCK-MDR1 efflux ratio 0.159 High 0.86
mh-clint Mouse hepatocyte intrinsic clearance 0.682 High 0.86
mppb Mouse plasma protein binding (% unbound) 89.220 High 0.86
nih-mdck-mdr1-efflux NIH MDCK-MDR1 efflux ratio 0.305 High 0.86
pfas-class PFAS structural alert (1 = True, 0 = False) 0
pppb Pig plasma protein binding (% unbound) 85.880 % High 0.86
rh-clint Rat hepatocyte intrinsic clearance 1.510 uL/min/10^6 cells High 0.86
rh-fuinc Rat hepatocyte fraction unbound in incubation 92.990 % High 0.86
rppb Rat plasma protein binding (% unbound) 85.170 % High 0.86
solubility-dd Solubility at pH 7.4 in DMSO 758.578 uM High 0.86

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)

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
Renal impairment 58.09 54.99 20 149 110208 42510958

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

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

Related Drugs by ATC class:

A16AX Various alimentary tract and metabolism products 23 drugs
anethole trithionedoxecitinedoxribtimineeliglustatfosdenopteringivosiranglycerol phenylbutyratelonafarniblumasiranmiglustatnedosirannitisinone show all 23 →

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

DiseaseRelationSNOMED_IDDOID
Fabry's disease indication 16652001 DOID:14499




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

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)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10383864 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10406143 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11241422 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9000011 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9480682 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9987263 May 16, 2027 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10525045 April 28, 2028 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10925866 April 28, 2028 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11033538 April 28, 2028 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12599595 April 28, 2028 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9999618 April 28, 2028 A METHOD OF REDUCING LEFT VENTRICULAR MASS INDEX (LVMI) IN A FABRY PATIENT BY ADMINISTERING MIGALASTAT
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9999618 April 28, 2028 A METHOD OF REDUCING PODOCYTE GLOBOTRIAOSYLCERAMIDE (GL-3) IN A FABRY PATIENT BY ADMINISTERING MIGALASTAT
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10813921 Feb. 12, 2029 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 8592362 Feb. 12, 2029 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 9095584 Feb. 12, 2029 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL RE48608 Oct. 20, 2031 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12599594 July 19, 2036 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10076514 March 15, 2037 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11234972 March 15, 2037 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10251873 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10471053 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10792278 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10792279 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10799491 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10806727 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10849889 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10849890 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10857141 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10857142 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10874655 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10874656 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 10874657 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11278536 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11278537 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11278538 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11278539 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11278540 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11357761 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11357762 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11357763 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11389436 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11389437 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11458128 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11666564 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11813255 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12042489 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12042490 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12109205 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12280042 May 30, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11903938 Aug. 17, 2038 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11357784 Feb. 6, 2039 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11642334 Feb. 20, 2039 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11633388 March 25, 2039 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 12594268 May 23, 2041 THE TREATMENT OF FABRY PATIENTS
EQ 123MG BASE GALAFOLD AMICUS THERAP US N208623 Aug. 10, 2018 RX CAPSULE ORAL 11833164 Jan. 11, 2042 THE TREATMENT OF FABRY PATIENTS

Orange Book exclusivity data (new drug applications)

None

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
Glycogen debranching enzyme Enzyme IC50 5 CHEMBL
Lactase-phlorizin hydrolase Enzyme IC50 4.30 CHEMBL
Maltase-glucoamylase, intestinal Enzyme IC50 5.82 CHEMBL
Lysosomal alpha-glucosidase Enzyme IC50 5.22 CHEMBL
Non-lysosomal glucosylceramidase Enzyme IC50 4 CHEMBL
Sucrase-isomaltase, intestinal Enzyme IC50 6.59 CHEMBL
Beta-galactosidase Enzyme Ki 4.90 CHEMBL
Lactase-phlorizin hydrolase Enzyme IC50 4.14 CHEMBL
Alpha-galactosidase C Enzyme IC50 5.74 CHEMBL
Alpha-galactosidase Enzyme IC50 8.52 CHEMBL
Beta-galactosidase Enzyme IC50 4.62 CHEMBL
Beta-galactosidase Enzyme Ki 4.89 CHEMBL

External reference:

IDSource
C4XNY919FW UNII
75172-81-5 SECONDARY_CAS_RN
D10359 KEGG_DRUG
4038521 VANDF
C2698220 UMLSCUI
DGJ PDB_CHEM_ID
CHEMBL110458 ChEMBL_ID
176077 PUBCHEM_CID
DB05018 DRUGBANK_ID
CHEMBL2107355 ChEMBL_ID
8700 INN_ID
C090092 MESH_SUPPLEMENTAL_RECORD_UI
10200 IUPHAR_LIGAND_ID
2054252 RXNORM
261328 MMSL
33035 MMSL
d08640 MMSL
017003 NDDF
017004 NDDF
765340000 SNOMEDCT_US
765341001 SNOMEDCT_US
773543004 SNOMEDCT_US
CHEBI:135923 CHEBI
C4XNY919FW INXIGHT DRUGS

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Galafold HUMAN PRESCRIPTION DRUG LABEL 1 71904-100 CAPSULE 123 mg ORAL NDA 27 sections