Stereochemistry | ABSOLUTE |
Molecular Formula | C6H13NO4.ClH |
Molecular Weight | 199.633 |
Optical Activity | ( + ) |
Defined Stereocenters | 4 / 4 |
E/Z Centers | 0 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
InChI
InChIKey=ZJIHMALTJRDNQI-OLALXQGDSA-N
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
Molecular Formula | ClH |
Molecular Weight | 36.461 |
Charge | 0 |
Count |
MOL RATIO
1 MOL RATIO (average) |
Stereochemistry | ACHIRAL |
Additional Stereochemistry | No |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Optical Activity | NONE |
Molecular Formula | C6H13NO4 |
Molecular Weight | 163.1717 |
Charge | 0 |
Count |
MOL RATIO
1 MOL RATIO (average) |
Stereochemistry | ABSOLUTE |
Additional Stereochemistry | No |
Defined Stereocenters | 4 / 4 |
E/Z Centers | 0 |
Optical Activity | UNSPECIFIED |
Migalastat (Galafold)-a small molecule drug developed by Amicus Therapeutics that restores the activity of specific mutant forms of α-galactosidase-has been approved for the treatment of Fabry disease in the EU in patients with amenable mutations. Migalastat attaches to certain unstable forms of alpha-galactosidase A, stabilising the enzyme. This allows the enzyme to be transported into areas of the cell where it can break down GL-3. Under the trade name Galafold (formerly known as Amigal), Migalastat is used to treat patients aged 16 years or over with Fabry disease. Because the number of patients with Fabry disease is low, the disease is considered ‘rare’, and the US Food and Drug Administration (FDA) assigned Galafold orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006.
Originator
Approval Year
AUC
Doses
AEs
Overview
CYP3A4 | CYP2C9 | CYP2D6 | hERG |
---|---|---|---|
Drug as perpetrator
Drug as victim
Tox targets
Sourcing
PubMed
Patents
Sample Use Guides
The recommended dosage regimen in adults and adolescents 16 years and older is 123 mg migalastat
(1 capsule) once every other day at the same time of day.
Route of Administration:
Oral
Migalastat (1-Deoxygalactonojirimycin) was the most potent inhibitor of alpha-Gal A in Fabry lymphoblasts with an IC50 value of 0.04 uM. Inclusion of 1-deoxygalactonojirimycin at 100 uM in culture medium of Fabry lymphoblasts increased the intracellular alpha-Gal A activity by 14-fold.