Migalastat

    WARNING: This product is for research use only, not for human or veterinary use.

MedKoo CAT#: 573849

CAS#: 108147-54-2 (free base)

Description: Migalastat is a potent inhibitor of glycolipid biosynthesis.


Chemical Structure

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Migalastat
CAS# 108147-54-2 (free base)

Theoretical Analysis

MedKoo Cat#: 573849
Name: Migalastat
CAS#: 108147-54-2 (free base)
Chemical Formula: C6H13NO4
Exact Mass: 163.08
Molecular Weight: 163.173
Elemental Analysis: C, 44.17; H, 8.03; N, 8.58; O, 39.22

Price and Availability

This product is not in stock, which may be available by custom synthesis. For cost-effective reason, minimum order is 1g (price is usually high, lead time is 2~3 months, depending on the technical challenge). Quote less than 1g will not be provided. To request quote, please email to sales @medkoo.com or click below button.
Note: Price will be listed if it is available in the future.

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Related CAS #: 108147-54-2 (free base)   75172-81-5 (HCl)  

Synonym: Amigal, DDIG, Migalastat; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; AT1001; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; Galafold

IUPAC/Chemical Name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol

InChi Key: LXBIFEVIBLOUGU-DPYQTVNSSA-N

InChi Code: 1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1

SMILES Code: [C@@H]1([C@H]([C@H](NC[C@@H]1O)CO)O)O

Appearance: Solid powder

Purity: >98% (or refer to the Certificate of Analysis)

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility: Soluble in DMSO

Shelf Life: >3 years if stored properly

Drug Formulation: This drug may be formulated in DMSO

Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code: 2934.99.9001

More Info:

Product Data:
Biological target: Migalastat (GR181413A free base) is an orally active and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM.
In vitro activity: TBD
In vivo activity: Western blots of the proteins indicated that there was an increase in stability of WT α-Gal A in flies treated with 50 μM migalastat (Figure 2A,B). There was a 2.5- and a 3-fold increase in the total amount of the A156V α-Gal A following 10 μM and 50 μM chaperone treatment, respectively, and elevation in the lysosomal fraction of the mutant protein, indicating that migalastat is able to bind the protein and to assist in its folding and trafficking from the ER to the lysosomes (Figure 2C,D). Moreover, even in the A285D-expressing flies, there was a 2.5-fold increase in the total amount of α-Gal A, as well as appearance of a small lysosomal fraction, following 20 μM migalastat treatment (Figure 2E,F). Reference: Int J Mol Sci. 2020 Oct; 21(19): 7397. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7583893/

Preparing Stock Solutions

The following data is based on the product molecular weight 163.17 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL
Formulation protocol: 1. Braunstein H, Papazian M, Maor G, Lukas J, Rolfs A, Horowitz M. Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological Chaperone Migalastat. Int J Mol Sci. 2020 Oct 7;21(19):7397. doi: 10.3390/ijms21197397. PMID: 33036426; PMCID: PMC7583893.
In vitro protocol: TBD
In vivo protocol: 1. Braunstein H, Papazian M, Maor G, Lukas J, Rolfs A, Horowitz M. Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological Chaperone Migalastat. Int J Mol Sci. 2020 Oct 7;21(19):7397. doi: 10.3390/ijms21197397. PMID: 33036426; PMCID: PMC7583893.

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1: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012-. Available from http://www.ncbi.nlm.nih.gov/books/NBK548736/ PubMed PMID: 31644045.

2: McCafferty EH, Scott LJ. Migalastat: A Review in Fabry Disease. Drugs. 2019 Apr;79(5):543-554. doi: 10.1007/s40265-019-01090-4. Review. Erratum in: Drugs. 2019 Jul 10;:. PubMed PMID: 30875019; PubMed Central PMCID: PMC6647464.

3: Migalastat for Fabry disease. Aust Prescr. 2019 Feb;42(1):36-37. doi: 10.18773/austprescr.2018.073. Epub 2018 Dec 13. Review. PubMed PMID: 30765910; PubMed Central PMCID: PMC6370600.

4: Pharmacoeconomic Review Report: Migalastat (Galafold): (Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb. Available from http://www.ncbi.nlm.nih.gov/books/NBK533451/ PubMed PMID: 30457791.

5: CADTH Canadian Drug Expert Committee Recommendation: Migalastat (Galafold — Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Jan. No abstract available. Available from http://www.ncbi.nlm.nih.gov/books/NBK533471/ PubMed PMID: 30457790.

6: Clinical Review Report: Migalastat (Galafold): (Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb. Available from http://www.ncbi.nlm.nih.gov/books/NBK533676/ PubMed PMID: 30457788.

7: Drugs and Lactation Database (LactMed) [Internet]. Bethesda (MD): National Library of Medicine (US); 2006-. Available from http://www.ncbi.nlm.nih.gov/books/NBK532501/ PubMed PMID: 30372005.