Migalastat is a small-molecule pharmacological chaperone used for the treatment of Fabry disease in patients with amenable GLA variants. It is a synthetic iminosugar that binds selectively and reversibly to certain mutant forms of alpha-galactosidase A (α-Gal A), helping stabilize the enzyme and facilitate its trafficking to lysosomes.
Migalastat is commonly supplied as migalastat hydrochloride, the hydrochloride salt of the active moiety. The FDA identifies migalastat hydrochloride as the active pharmaceutical ingredient in the approved oral product.
Migalastat is a specialized rare-disease / metabolic-disorder pharmaceutical API intended for pharmaceutical formulation and research. The molecule requires stringent control of stereochemical identity, assay, related substances, water content and other quality attributes.
CAS Number (Migalastat): 108147-54-2
CAS Number (Migalastat Hydrochloride): 75172-81-5
Molecular Formula: C6H13NO4
Molecular Weight: 163.17 g/mol
Hydrochloride Formula: C6H14ClNO4
Hydrochloride Molecular Weight: 199.63 g/mol
Chemical Name: (2R,3S,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol
Synonyms: 1-Deoxygalactonojirimycin, 1-Deoxygalactonojirimycin Hydrochloride
Drug Class: Pharmacological Chaperone
Therapeutic Area: Rare Diseases / Metabolic Disorders
Molecule Type: Synthetic Small Molecule
The FDA confirms the absolute stereochemistry of migalastat and identifies four defined stereocenters.
Migalastat acts as a pharmacological chaperone for certain amenable mutant forms of α-Gal A.
Its mechanism involves:
Migalastat API is primarily associated with:
Migalastat hydrochloride is formulated as an oral capsule. The FDA-approved capsule contains 123 mg of migalastat, equivalent to 150 mg of migalastat hydrochloride.
Migalastat hydrochloride is described by the FDA as a white to almost white crystalline solid. It is freely soluble in aqueous media within the pH range of 1.2 to 7.5.
A controlled Migalastat API manufacturing process may involve:
Typical Migalastat API testing may include:
Store Migalastat API in a tightly closed pharmaceutical container under controlled storage conditions, protected from excessive moisture, heat and direct light. Final storage requirements should follow the applicable API specification and stability data.
Migalastat is relevant to research involving:
Migalastat is a pharmacological chaperone used for Fabry disease in patients with amenable GLA variants. Migalastat has CAS 108147-54-2, molecular formula C6H13NO4, and molecular weight 163.17 g/mol. Its hydrochloride salt has CAS 75172-81-5, formula C6H14ClNO4, and molecular weight 199.63 g/mol.
| Parameter | Specification |
|---|---|
| Appearance | White crystalline powder or crystals |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |