Migalastat

Identification

Name
Migalastat
Accession Number
DB05018
Type
Small Molecule
Groups
Approved, Investigational
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
Synonyms
Not Available
Product Ingredients
IngredientUNIICASInChI Key
Migalastat hydrochlorideCLY7M0XD2075172-81-5ZJIHMALTJRDNQI-OLALXQGDSA-N
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
GalafoldCapsule123 mgOralAmicus TherapeuticsNot applicableNot applicableCanada
International/Other Brands
Amigal
Categories
UNII
C4XNY919FW
CAS number
Not Available
Weight
Average: 163.1717
Monoisotopic: 163.084457909
Chemical Formula
C6H13NO4
InChI Key
LXBIFEVIBLOUGU-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

Pharmacology

Indication

Investigated for use/treatment in fabry disease.

Structured Indications
Pharmacodynamics
Not 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.

TargetActionsOrganism
UAlpha-galactosidase ANot AvailableHuman
Absorption
Not Available
Volume of distribution
Not Available
Protein binding
Not Available
Metabolism
Not Available
Route of elimination
Not Available
Half life
Not Available
Clearance
Not Available
Toxicity
Not Available
Affected organisms
Not Available
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
Not Available
Food Interactions
Not Available

References

General References
Not Available
External Links
PubChem Compound
176077
PubChem Substance
347827704
ChemSpider
153388
BindingDB
50163440
ChEBI
135923
ChEMBL
CHEMBL110458
HET
DGJ
AHFS Codes
  • 92:92.00 — Other Miscellaneous Therapeutic Agents
PDB Entries
3s5y / 3thd / 3tv8 / 4cu8 / 4d1j / 4do5 / 4fns / 4ufm

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1CompletedTreatmentFabry's Disease3
2CompletedTreatmentFabry's Disease5
3Active Not RecruitingTreatmentFabry's Disease1
3CompletedTreatmentFabry's Disease3
Not AvailableAvailableNot AvailableFabry's Disease1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage forms
FormRouteStrength
CapsuleOral123 mg
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not 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 features
Not Available

Spectra

Mass Spec (NIST)
Not Available
Spectra
SpectrumSpectrum TypeSplash Key
Predicted GC-MS Spectrum - GC-MSPredicted GC-MSNot Available
Predicted MS/MS Spectrum - 10V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Positive (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 10V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 20V, Negative (Annotated)Predicted LC-MS/MSNot Available
Predicted MS/MS Spectrum - 40V, Negative (Annotated)Predicted LC-MS/MSNot Available

Taxonomy

Description
This compound belongs to the class of organic compounds known as piperidines. These are compounds containing a piperidine ring, which is a saturated aliphatic six-member ring with one nitrogen atom and five carbon atoms.
Kingdom
Organic compounds
Super Class
Organoheterocyclic compounds
Class
Piperidines
Sub Class
Not Available
Direct Parent
Piperidines
Alternative Parents
Secondary alcohols / 1,2-aminoalcohols / Polyols / Dialkylamines / Azacyclic compounds / Primary alcohols / Organopnictogen compounds / Hydrocarbon derivatives
Substituents
Piperidine / 1,2-aminoalcohol / Secondary alcohol / Secondary aliphatic amine / Polyol / Secondary amine / Azacycle / Primary alcohol / Alcohol / Organic oxygen compound
Molecular Framework
Aliphatic heteromonocyclic compounds
External Descriptors
Not Available

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 January 15, 2018 08:55