Migalastat Hydrochloride is a small-molecule pharmacological chaperone and hydrochloride salt of migalastat. It is used in the development and manufacture of pharmaceutical formulations for the treatment of Fabry disease in patients with amenable mutations in the α-galactosidase A gene.
Migalastat is an iminosugar that binds selectively and reversibly to certain mutant forms of α-galactosidase A (α-Gal A). This interaction can help stabilize the enzyme and promote its trafficking to lysosomes, where it can contribute to the breakdown of accumulated globotriaosylceramide and related substrates.
Migalastat Hydrochloride is a specialized rare-disease and metabolic-disorder pharmaceutical API used for oral pharmaceutical formulation development.
Important API quality attributes may include identity, assay, related substances, water content, residual solvents, elemental impurities, optical purity and other product-specific analytical parameters.
CAS Number: 75172-81-5
Molecular Formula: C₆H₁₃NO₄·HCl
Molecular Weight: 199.63 g/mol
Chemical Name: (+)-(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride
Active Moiety: Migalastat
Drug Class: Pharmacological Chaperone
Therapeutic Class: Treatment of Fabry Disease
Therapeutic Area: Rare Diseases / Metabolic Disorders
Molecule Type: Small-Molecule Iminosugar
Migalastat Hydrochloride acts as a pharmacological chaperone for α-galactosidase A.
Its pharmacological activity includes:
Migalastat binds to the active site of certain mutant α-Gal A enzymes and stabilizes them, helping the enzyme reach the lysosome and retain activity.
Migalastat Hydrochloride API is used in pharmaceutical development for:
Migalastat Hydrochloride is particularly relevant to Fabry disease, a lysosomal storage disorder associated with deficient or dysfunctional α-galactosidase A.
Migalastat is used for patients with Fabry disease who have amenable mutations, where the pharmacological chaperone mechanism can increase the functional activity of the patient's endogenous α-Gal A enzyme.
Migalastat Hydrochloride is described 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.
The crystalline form, water content, purity and other physicochemical characteristics should be controlled according to the applicable API specification.
A controlled Migalastat Hydrochloride API manufacturing process may involve:
The final process and controls should follow the approved manufacturing process and applicable pharmaceutical quality requirements.
Typical Migalastat Hydrochloride API testing may include:
Final numerical acceptance criteria should be taken from the current validated API specification or CoA rather than using generic limits.
Store Migalastat Hydrochloride API in a tightly closed container under controlled pharmaceutical storage conditions, protected from excessive moisture, heat and direct light.
For reference, the approved pharmaceutical product is stored at 20–25°C, with permitted excursions between 15–30°C; API storage should ultimately follow the validated stability data and approved API specification.
Migalastat Hydrochloride is relevant to research involving:
Migalastat Hydrochloride is a small-molecule pharmacological chaperone associated with the treatment of Fabry disease in patients with amenable α-galactosidase A mutations. It has CAS Number 75172-81-5, molecular formula C₆H₁₃NO₄·HCl, 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 2.0% |
| Residue on Ignition | NMT 0.5% |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |