Migalastat Hydrochloride

Base Information

• Chemical Name: Migalastat Hydrochloride
• CAS No.: 75172-81-5
• Molecular Formula: C₆H₁₃NO₄*ClH
• Molecular Weight: 199.634
• Hs Code.: 29333990
• UNII: CLY7M0XD20
• ChEMBL ID: CHEMBL2107355
• DSSTox Substance ID: DTXSID801026249
• NCI Thesaurus Code: C78121
• RXCUI: 2054264
• Wikidata: Q27275527
• Mol file: 75172-81-5.mol

Synonyms:1-butyl-2-(hydroxylmethyl)piperidine-3,4,5-triol;1-deoxy-galactonojirimycin;1-deoxygalacto-nojirimycin;1-deoxygalactonojirimycin;AT1001 deoxyjirimycin;Galafold;GR181413A;lucerastat;migalastat;migalastat HCl;migalastat hydrochloride;N-butyldeoxygalacto-nojirimycin;N-butyldeoxygalactonojirimycin;NB-DGJ

Chemical Property of Migalastat Hydrochloride

Chemical Property:

• Vapor Pressure: 1.98E-07mmHg at 25°C
• Melting Point: 260 °C
• Boiling Point: 382.7 °C at 760 mmHg
• Flash Point: 185.2 °C
• Storage Temp.: 2-8°C
• Solubility.: Methanol (Slightly), Water (Slightly)
• Hydrogen Bond Donor Count: 6
• Hydrogen Bond Acceptor Count: 5
• Rotatable Bond Count: 1
• Exact Mass: 199.0611356
• Heavy Atom Count: 12
• Complexity: 132

Purity/Quality:

99% ^*data from raw suppliers

Deoxygalactonojirimycin, Hydrochloride ^*data from reagent suppliers

Safty Information:

• Pictogram(s): Xi
• Hazard Codes: Xi
• Statements: 36/37/38
• Safety Statements: 26-36

MSDS Files:

SDS file from LookChem

Useful:

• Canonical SMILES: C1C(C(C(C(N1)CO)O)O)O.Cl
• Isomeric SMILES: C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O.Cl
• Recent ClinicalTrials: Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
• Recent EU Clinical Trials: A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy
• Description: Migalastat, which is marketed by Amicus Therapeutics, received approval in the EU for the treatment of Fabry disease in adults and adolescents aged 16 or older. Fabry disease is caused by mutations of the enzyme α-galactosidase A (α-GAL A) that cause protein misfolding and prevents efficient metabolism of the glycosphingolipid globotriaosylceramide (GL3). Accumulation of GL3 in lysosomes, blood vessels, and various tissues ultimately leads to significant heart, kidney, and dermatological problems. Migalastat functions as a molecular chaperone to α- GAL A, engaging the enzyme and enabling it to adopt the proper conformation allowing for efficient breakdown of GL3. Because the standard of care prior to 2016 for treating Fabry disease was enzyme replacement therapy (ERT), migalastat’s approval in the EU represents an important advance for patients suffering from this disorder.
• Uses: Proven to be an extremely potent and selective a-D-galactosidase inhibitor. Deoxygalactonojirimycin hydrochloride has been used as an α-galactosidase A inhibitor to assess the enzymatic activity of α-galactosidase A. It has also been used as an α-galactosidase A inhibitor to study its effects on the mRNA levels in human embryonic kidney (HEK) cells and hippocampal neurons.

Technology Process of Migalastat Hydrochloride

There total 1 articles about Migalastat Hydrochloride which guide to synthetic route it. The literature collected by LookChem mainly comes from the sharing of users and the free literature resources found by Internet computing technology. We keep the original model of the professional version of literature to make it easier and faster for users to retrieve and use. At the same time, we analyze and calculate the most feasible synthesis route with the highest yield for your reference as below:

synthetic route:

Reference yield: 16.0%

5-azido-5-deoxy-D-glucofuranose (146897-25-8) → 1,5-dideoxy-1,5-imino-D-glucitol (19130-96-2) + 1-deoxynojirimycin hydrochloride (73285-50-4,73465-43-7,75172-81-5,118464-54-3,126663-84-1,126923-63-5,136658-34-9)

Guidance literature:

5-azido-5-deoxy-D-glucofuranose; With palladium 10% on activated carbon; hydrogen; In water; at 20 ℃; under 2068.65 - 2585.81 Torr;

With hydrogenchloride; In water; pH=2;

DOI:10.1016/j.tetasy.2010.01.017

Reference yield: 86.0%

benzyloxyacetoaldehyde (60656-87-3) + 1-deoxynojirimycin hydrochloride (73285-50-4,73465-43-7,75172-81-5,118464-54-3,126663-84-1,126923-63-5,136658-34-9) → N-(2-benzyloxyethyl)-1-deoxynojirimycin (1203659-54-4)

Guidance literature:

With sodium cyanoborohydride; acetic acid; In methanol; at 20 ℃; for 48h;

DOI:10.1021/jm901281m

Reference yield: 80.8%

1-deoxynojirimycin hydrochloride (73285-50-4,73465-43-7,75172-81-5,118464-54-3,126663-84-1,126923-63-5,136658-34-9) + benzyl chloroformate (501-53-1,94274-21-2) → N-Benzyloxycarbonyl-1,5-dideoxy-1,5-imino-D-glucitol (130539-12-7)

Guidance literature:

With sodium hydrogencarbonate; In toluene; at 0 - 20 ℃; for 6.25h;

upstream raw materials:

5-azido-5-deoxy-D-glucofuranose

Downstream raw materials:

N-(2-benzyloxyethyl)-1-deoxynojirimycin

1,5-dideoxy-1,5-imino-D-glucitol

Refernces