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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

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.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
0.04 µM [IC50]
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Galafold
PubMed

PubMed

TitleDatePubMed
[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
Patents

Patents

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.
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