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Migalastat  (Synonyms: GR181413A free base; 1-Deoxygalactonojirimycin)

Cat. No.: HY-14929
Handling Instructions

Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity.

At equivalent molar concentrations, both the salt and free forms of a compound exhibit comparable biological activity. Nevertheless, the salt form (Migalastat hydrochloride) usually boasts enhanced water solubility and stability.

For research use only. We do not sell to patients.

Migalastat Chemical Structure

Migalastat Chemical Structure

CAS No. : 108147-54-2

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  • Biological Activity

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Description

Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity[1].

IC50 & Target

IC50: 0.04 μM (human α-Gal A)[1];
Ki: 0.04 μM (human α-Gal A)[1]

Cellular Effect
Cell Line Type Value Description References
Lymphocyte CC50
> 200 μM
Compound: DGJ
Cytotoxicity against human lymphocytes measured after 48 to 72 hrs by alamar blue assay
Cytotoxicity against human lymphocytes measured after 48 to 72 hrs by alamar blue assay
[PMID: 27744182]
Lymphocyte CC50
> 200 μM
Compound: 2; DGJ
Cytotoxicity against human lymphocytes assessed as reduction in cell viability after 48 to 72 hrs by alamar blue assay
Cytotoxicity against human lymphocytes assessed as reduction in cell viability after 48 to 72 hrs by alamar blue assay
[PMID: 27474919]
In Vitro

Migalastat inhibits human lysosomal a-Gal A with IC50 and Ki values of 0.04 μM[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Cell Viability Assay[4]

Cell Line: EHK cells mutated α-Gal A
Concentration: 10 μM
Incubation Time: 9 days
Result: Reduced Gb3 accumulation and lysosome volume.
In Vivo

Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A[2].
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM)[2].
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment[2].
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively[3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Male nontransgenic (Non-Tg) C57BL/6 mice; transgenic mice expressing human mutant R301Q α-Gal A (TgM), α-Gal A knockout mice (KO), mice express human R301Q α-Gal A in a null background (TgM/KO)[2]
Dosage: 3 mg/kg
Administration: Oral gavage; every day for 4 weeks
Result: Reduced Globotriaosylceramide (Gb3) storage remarkably in kidney of mice.
Clinical Trial
Molecular Weight

163.17

Formula

C6H13NO4

CAS No.
SMILES

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

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

Please store the product under the recommended conditions in the Certificate of Analysis.

Purity & Documentation
References
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Migalastat Related Classifications

  • Molarity Calculator

  • Dilution Calculator

The molarity calculator equation

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass   Concentration   Volume   Molecular Weight *
= × ×

The dilution calculator equation

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
× = ×
C1   V1   C2   V2
Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Migalastat
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HY-14929
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