Identification
NameMigalastat
Accession NumberDB05018
TypeSmall Molecule
GroupsInvestigational
Description

Amigal (migalastat hydrochloride) is an experimental, oral therapy for the treatment of Fabry disease and belongs to a class of molecules known as pharmacological chaperones.

Structure
Thumb
SynonymsNot Available
External IDs Not Available
Product Ingredients
IngredientUNIICASInChI KeyDetails
Migalastat hydrochlorideCLY7M0XD20 75172-81-5ZJIHMALTJRDNQI-OLALXQGDSA-NDetails
Approved Prescription ProductsNot Available
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International Brands
NameCompany
AmigalNot Available
Brand mixturesNot Available
Categories
UNIIC4XNY919FW
CAS numberNot Available
WeightAverage: 163.1717
Monoisotopic: 163.084457909
Chemical FormulaC6H13NO4
InChI KeyLXBIFEVIBLOUGU-DPYQTVNSSA-N
InChI
InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1
IUPAC Name
(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
SMILES
[H][C@]1(O)CN[C@]([H])(CO)[C@]([H])(O)[C@]1([H])O
Pharmacology
Indication

Investigated for use/treatment in fabry disease.

Structured Indications Not Available
PharmacodynamicsNot Available
Mechanism of action

Amigal acts by selectively binding to the misfolded enzyme responsible for Fabry disease, α-GAL. This may increase the enzyme’s stability and promote the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced and the enzyme can perform its normal biological function, which is the breakdown of its natural substrate, GL-3.

TargetKindPharmacological actionActionsOrganismUniProt ID
Alpha-galactosidase AProteinunknownNot AvailableHumanP06280 details
Related Articles
AbsorptionNot Available
Volume of distributionNot Available
Protein bindingNot Available
MetabolismNot Available
Route of eliminationNot Available
Half lifeNot Available
ClearanceNot Available
ToxicityNot Available
Affected organismsNot Available
PathwaysNot Available
Pharmacogenomic Effects/ADRs Not Available
Interactions
Drug Interactions Not Available
Food InteractionsNot Available
References
Synthesis ReferenceNot Available
General ReferencesNot Available
External Links
ATC CodesNot Available
AHFS CodesNot Available
PDB EntriesNot Available
FDA labelNot Available
MSDSNot Available
Clinical Trials
Clinical Trials
PhaseStatusPurposeConditionsCount
1CompletedTreatmentFabry's Disease3
2CompletedTreatmentFabry's Disease5
3Active Not RecruitingTreatmentFabry's Disease1
3AvailableNot AvailableFabry's Disease1
3CompletedTreatmentFabry's Disease3
Pharmacoeconomics
ManufacturersNot Available
PackagersNot Available
Dosage formsNot Available
PricesNot Available
PatentsNot Available
Properties
StateSolid
Experimental PropertiesNot Available
Predicted Properties
PropertyValueSource
Water Solubility511.0 mg/mLALOGPS
logP-2.2ALOGPS
logP-2.9ChemAxon
logS0.5ALOGPS
pKa (Strongest Acidic)12.91ChemAxon
pKa (Strongest Basic)8.06ChemAxon
Physiological Charge1ChemAxon
Hydrogen Acceptor Count5ChemAxon
Hydrogen Donor Count5ChemAxon
Polar Surface Area92.95 Å2ChemAxon
Rotatable Bond Count1ChemAxon
Refractivity36.57 m3·mol-1ChemAxon
Polarizability15.86 Å3ChemAxon
Number of Rings1ChemAxon
Bioavailability1ChemAxon
Rule of FiveYesChemAxon
Ghose FilterNoChemAxon
Veber's RuleNoChemAxon
MDDR-like RuleNoChemAxon
Predicted ADMET featuresNot Available
Spectra
Mass Spec (NIST)Not Available
SpectraNot Available
Taxonomy
ClassificationNot classified

Targets

Kind
Protein
Organism
Human
Pharmacological action
unknown
General Function:
Receptor binding
Specific Function:
Not Available
Gene Name:
GLA
Uniprot ID:
P06280
Uniprot Name:
Alpha-galactosidase A
Molecular Weight:
48766.34 Da
Drug created on October 21, 2007 16:23 / Updated on June 14, 2017 10:31