WARNING: This product is for research use only, not for human or veterinary use.
MedKoo CAT#: 510224
CAS#: 75172-81-5 (HCl)
Description: Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of α-Gal A. Oral administration of migalastat HCl reduces tissue GL-3 in Fabry transgenic mice, and in urine and kidneys of some FD patients. Migalastat HCl may provide a potential novel genotype-specific treatment for Fabry Disease (FD). Phase 3 studies are ongoing. Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues.
MedKoo Cat#: 510224
Name: Migalastat HCl
CAS#: 75172-81-5 (HCl)
Chemical Formula: C6H13NO4
Molecular Weight: 163.17
Elemental Analysis: C, 44.16; H, 8.03; N, 8.58; O, 39.22
Synonym: AT1001; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; 1,5-Dideoxy-1,5-imino-D-galactitol; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; Amigal; Migalastat; Galafold
IUPAC/Chemical Name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride.
InChi Key: ZJIHMALTJRDNQI-OLALXQGDSA-N
InChi Code: 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
SMILES Code: O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O.[H]Cl
Appearance: Grey to light yellow 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: 0 Â– 4 C for short term (weeks to 1 month) or -20 C for long terms (months to years).
Solubility: soluble in DMSO, soluble in water.
Shelf Life: >2 years if stored properly.
Drug Formulation: PBS buffer, water, etc.
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
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.
|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|
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.