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| 5mg |
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| 25mg |
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| 50mg |
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| 100mg |
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Purity: ≥98%
Migalastat hydrochloride (formerly GR-181413A; AT1001; GR181413A; AT-1001; Galafold), the hydrochloride salt of migalastate, is an inhibitor of α-galactosidase A (α-Gal A) and a medication approved by FDA in August 2018 to treat adults with Fabry disease. It is a pharmacological chaperone that potently and selectively binds, stabilizes, and increases cellular levels of α-Gal A with an IC50 of 0.04 μM for human 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. Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A (α-Gal A), and involves pathological accumulation of globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). Oral administration of migalastat HCl to transgenic mice reduced elevated lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively, generally equal to or greater than observed for GL-3. Furthermore, baseline plasma lyso-Gb3 levels were markedly elevated in six male FD patients enrolled in Phase 2 studies. Oral administration of migalastat HCl (150 mg QOD) reduced urine GL-3 and plasma lyso-Gb3 in three subjects (range: 15% to 46% within 48 weeks of treatment). In contrast, three showed no reductions in either substrate. These results suggest that measurement of tissue and/or plasma lyso-Gb3 is feasible and may be warranted in future studies of migalastat HCl or other new potential therapies for FD.
| ln Vitro |
Regarding human lysosomal a-Gal A, migasalstat hydrochloride (GR181413A) exhibits an IC50 and Ki value of 0.04 μM [1].
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| ln Vivo |
Insufficient α-galactosidase A activity is the cause of Fabry disease, a latent X-linked illness [2]. In splenic points expressing human mutant α-Gal A (TgM), migalastat (passage, 3 mg/kg daily for 4 weeks) increases cardiac, renal, and cardiac α-Gal A activity at dose and time of illumination [2]. Upon stopping Migalastat for two weeks, half-lives of all major concerns were less than one day [2]. Migalastat lowers FXR, cardiac, and skin solution-GB3 levels by 64%, 59%, and 81%, respectively, when taken lateral (100 mg/kg daily for 28 days) [3].
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| Cell Assay |
Cell Viability Assay [4]
Cell Types: EHK Cells Mutated α-Gal A Tested Concentrations: 10 μM Incubation Duration: 9 Days Experimental Results: Gb3 accumulation and lysosomal volume reduction. |
| Animal Protocol |
Animal/Disease Models: Male non-transgenic (non-transgenic) Tg)C57BL/6 mice; transgenic mice expressing human mutant R301Q α-Gal A (TgM), α-Gal A knockout mice (KO), on null background Mice expressing human R301Q α-Gal A (TgM/KO)[2]
Doses: 3 mg/kg Route of Administration: po (oral gavage); one time/day for 4 weeks Experimental Results: Triacylceramide (Gb3) in mouse kidneys storage is Dramatically diminished. |
| Toxicity/Toxicokinetics |
Effects During Pregnancy and Lactation
◉ Summary of Use during Lactation No information is available on the use of migalastat during breastfeeding. Because no information is available on the use of migalastat during breastfeeding caution should be used, especially while nursing a newborn or preterm infant. ◉ Effects in Breastfed Infants Relevant published information was not found as of the revision date. ◉ Effects on Lactation and Breastmilk Relevant published information was not found as of the revision date. |
| References |
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| Additional Infomation |
See also: Migalastat (has active moiety).
Drug Indication Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation. Treatment of Fabry disease |
| Molecular Formula |
C6H13NO4.HCL
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|---|---|
| Molecular Weight |
199.63266
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| Exact Mass |
199.061
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| CAS # |
75172-81-5
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| Related CAS # |
Migalastat;108147-54-2
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| PubChem CID |
11644097
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| Appearance |
White to light brown solid powder
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| Boiling Point |
382.7ºC at 760 mmHg
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| Melting Point |
260ºC
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| Flash Point |
185.2ºC
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| Hydrogen Bond Donor Count |
6
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| Hydrogen Bond Acceptor Count |
5
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| Rotatable Bond Count |
1
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| Heavy Atom Count |
12
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| Complexity |
132
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| Defined Atom Stereocenter Count |
4
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| SMILES |
C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O.Cl
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| InChi Key |
ZJIHMALTJRDNQI-OLALXQGDSA-N
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| 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
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| Chemical Name |
(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride.
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| Synonyms |
1-Deoxygalactonojirimycin hydrochloride; AT1001 HCl; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; 1,5-Dideoxy-1,5-imino-D-galactitol; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; Amigal; Migalastat; trade name: Galafold
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment, avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
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| Solubility (In Vivo) |
Solubility in Formulation 1: 100 mg/mL (500.93 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.
(Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 5.0093 mL | 25.0463 mL | 50.0927 mL | |
| 5 mM | 1.0019 mL | 5.0093 mL | 10.0185 mL | |
| 10 mM | 0.5009 mL | 2.5046 mL | 5.0093 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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