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Details

Stereochemistry ABSOLUTE
Molecular Formula C6H13NO4
Molecular Weight 163.1717
Optical Activity UNSPECIFIED
Defined Stereocenters 4 / 4
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of MIGALASTAT

SMILES

OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O

InChI

InChIKey=LXBIFEVIBLOUGU-DPYQTVNSSA-N
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

HIDE SMILES / InChI

Molecular Formula C6H13NO4
Molecular Weight 163.1717
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry
Defined Stereocenters 4 / 4
E/Z Centers 0
Optical Activity UNSPECIFIED

Description

Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Migalastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.

CNS Activity

Approval Year

Target Info

Target Info

Primary TargetPharmacologyConditionPotency
0.04 µM [IC50]
Condition Info

Condition Info

ConditionModalityTargetsHighest PhaseProduct
Primary
Galafold
PMID

PMID

TitleDatePMID
Interference with HIV-induced syncytium formation and viral infectivity by inhibitors of trimming glucosidase.
1987 Nov 5-11
Inhibition of HIV replication by amino-sugar derivatives.
1988 Sep 12
Attenuation of HIV-1 infectivity by an inhibitor of oligosaccharide processing.
1990 Jun
Inhibitory effect of novel 1-deoxynojirimycin derivatives on HIV-1 replication.
1990 Oct
6-0-butanoylcastanospermine (MDL 28,574) inhibits glycoprotein processing and the growth of HIVs.
1991 Jun
Inhibition of alpha-glucosidase I of the glycoprotein-processing enzymes by 6-O-butanoyl castanospermine (MDL 28,574) and its consequences in human immunodeficiency virus-infected T cells.
1994 Aug
N-alkylated nitrogen-in-the-ring sugars: conformational basis of inhibition of glycosidases and HIV-1 replication.
1995 Jun 23
Imino sugars that are less toxic but more potent as antivirals, in vitro, compared with N-n-nonyl DNJ.
2002 Sep
Glycosylation inhibitors and neuraminidase enhance human immunodeficiency virus type 1 binding and neutralization by mannose-binding lectin.
2003 Feb
Imino sugars are potent agonists of the human glucose sensor SGLT3.
2007 Feb
Inhibition of alpha-mannosidase attenuates endoplasmic reticulum stress-induced neuronal cell death.
2009 Jan
[Enzyme replacement therapy of lysosomal storage diseases].
2010 Dec
α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry disease mutants.
2012 Aug 17
1-deoxynojirimycin isolated from Bacillus subtilis improves hepatic lipid metabolism and mitochondrial function in high-fat-fed mice.
2015 Jan
Hypoglycemic effect of deoxynojirimycin-polysaccharide on high fat diet and streptozotocin-induced diabetic mice via regulation of hepatic glucose metabolism.
2015 Jan 5
Patent

Sample Use Guides

In Vivo Use Guide
The recommended dosage regimen in adults and adolescents 16 years and older is 123 mg migalastat (1 capsule) once every other day at the same time of day.
Route of Administration: Oral
In Vitro Use Guide
Migalastat (1-Deoxygalactonojirimycin) was the most potent inhibitor of alpha-Gal A in Fabry lymphoblasts with an IC50 value of 0.04 uM. Inclusion of 1-deoxygalactonojirimycin at 100 uM in culture medium of Fabry lymphoblasts increased the intracellular alpha-Gal A activity by 14-fold.
Substance Class Chemical
Created
by admin
on Tue Mar 06 17:09:50 UTC 2018
Edited
by admin
on Tue Mar 06 17:09:50 UTC 2018
Record UNII
C4XNY919FW
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
MIGALASTAT
INN   USAN   WHO-DD  
INN   USAN  
Official Name English
MIGALASTAT [WHO-DD]
Common Name English
MIGALASTAT [INN]
Common Name English
MIGALASTAT [USAN]
Common Name English
Code System Code Type Description
CAS
108147-54-2
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY
PUBCHEM
176077
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY SWITZERF
ChEMBL
CHEMBL110458
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY
EVMPD
SUB177094
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY
INN
8700
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY
NCI_THESAURUS
C78120
Created by admin on Tue Mar 06 17:09:50 UTC 2018 , Edited by admin on Tue Mar 06 17:09:50 UTC 2018
PRIMARY
Related Record Type Details
ACTIVE MOIETY