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Details

Stereochemistry ABSOLUTE
Molecular Formula C6H13NO4.ClH
Molecular Weight 199.633
Optical Activity ( + )
Defined Stereocenters 4 / 4
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of MIGALASTAT HYDROCHLORIDE

SMILES

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

InChI

InChIKey=ZJIHMALTJRDNQI-OLALXQGDSA-N
InChI=1S/C6H13NO4.ClH/c8-2-3-5(10)6(11)4(9)1-7-3;/h3-11H,1-2H2;1H/t3-,4+,5+,6-;/m1./s1

HIDE SMILES / InChI

Molecular Formula ClH
Molecular Weight 36.461
Charge 0
Count
MOL RATIO 1 MOL RATIO (average)
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Molecular Formula C6H13NO4
Molecular Weight 163.1717
Charge 0
Count
MOL RATIO 1 MOL RATIO (average)
Stereochemistry ABSOLUTE
Additional Stereochemistry No
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.

Originator

Approval Year

Targets

Primary TargetPharmacologyConditionPotency
0.04 µM [IC50]

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Galafold

Cmax

ValueDoseCo-administeredAnalytePopulation
2029 ng/mL
150 mg 1 times / 2 days steady-state, oral
MIGALASTAT plasma
Homo sapiens
1323 ng/mL
150 mg single, oral
MIGALASTAT plasma
Homo sapiens
1561 ng/mL
150 mg single, oral
MIGALASTAT plasma
Homo sapiens

AUC

ValueDoseCo-administeredAnalytePopulation
10638 ng × h/mL
150 mg 1 times / 2 days steady-state, oral
MIGALASTAT plasma
Homo sapiens
6132 ng × h/mL
150 mg single, oral
MIGALASTAT plasma
Homo sapiens
9805 ng × h/mL
150 mg single, oral
MIGALASTAT plasma
Homo sapiens
10637.9 ng*h/mL
150 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens
13217.2 ng*h/mL
250 mg single, oral
MIGALASTAT plasma
Homo sapiens
14850.6 ng*h/mL
250 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens
2300.3 ng*h/mL
50 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens
2628.9 ng*h/mL
50 mg single, oral
MIGALASTAT plasma
Homo sapiens
3191.6 ng*h/mL
50 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens
8581.9 ng*h/mL
150 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens
8941.6 ng*h/mL
150 mg single, oral
MIGALASTAT plasma
Homo sapiens
9970.3 ng*h/mL
250 mg 1 times / 2 days steady, oral
MIGALASTAT plasma
Homo sapiens

T1/2

ValueDoseCo-administeredAnalytePopulation
4.9 h
150 mg single, oral
MIGALASTAT plasma
Homo sapiens
3.9 h
150 mg single, oral
MIGALASTAT plasma
Homo sapiens

Funbound

ValueDoseCo-administeredAnalytePopulation
100%
150 mg 1 times / 2 days steady-state, oral
MIGALASTAT plasma
Homo sapiens

Doses

AEs

Drug as perpetrator​

Drug as victim

Tox targets

Sourcing

PubMed

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.
Substance Class Chemical
Record UNII
CLY7M0XD20
Record Status Validated (UNII)
Record Version