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Galafold Generic Name & Formulations
Legal Class
Rx
General Description
Migalastat 123mg; caps.
Pharmacological Class
Alpha-galactosidase A pharmacological chaperone.
How Supplied
Caps—14
Manufacturer
Generic Availability
NO
Mechanism of Action
Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH and at a higher concentration of relevant substrates, migalastat dissociates from alpha-Gal A allowing it to break down the glycosphingolipids globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3).
Galafold Indications
Indications
Treatment of adults with confirmed Fabry disease and an amenable GLA variant based on in vitro assay data.
Galafold Dosage and Administration
Adult
Swallow whole. Take on empty stomach. Avoid food or caffeine ≥2hrs before and 2hrs after dose. 123mg once every other day; do not take on 2 consecutive days.
Children
Not established.
Galafold Contraindications
Not Applicable
Galafold Boxed Warnings
Not Applicable
Galafold Warnings/Precautions
Warnings/Precautions
Severe renal impairment or ESRD on dialysis: not recommended. Pregnancy. Nursing mothers.
Galafold Pharmacokinetics
Absorption
Absolute bioavailability (AUC): ~75% (after a single Galafold 123mg dose). Time to peak plasma concentration (tmax): ~3 hours.
Distribution
Apparent volume of distribution (Vz/F) at steady state: ~89 L (range: 77–133 L).
Elimination
Renal (~77%), fecal (20%). Half-life: ~4 hours.
Galafold Interactions
Interactions
May be antagonized by caffeine; separate by at least 2hrs before and 2hrs after Galafold administration.
Galafold Adverse Reactions
Adverse Reactions
Headache, nasopharyngitis, urinary tract infection, nausea, pyrexia, abdominal pain, back pain, cough, diarrhea, epistaxis.
Galafold Clinical Trials
See Literature
Galafold Note
Not Applicable
Galafold Patient Counseling
See Literature
