Note: Determine presence of an amenable galactosidase alpha (GLA) gene variant prior to therapy.
Fabry disease (with amenable GLA gene variant): Oral: 123 mg once every other day (do not administer on 2 consecutive days).
Missed doses: If a dose is missed entirely for the day, administer the missed dose only if it is within 12 hours of the normal time that the dose should have been administered. If more than 12 hours have passed, resume at the next planned dosing day and time, according to the every-other-day dosing schedule. Do not administer on 2 consecutive days.
eGFR ≥30 mL/minute/1.73 m2: No dosage adjustment necessary.
eGFR <30 mL/minute/1.73 m2: Use is not recommended (has not been studied).
ESRD requiring dialysis: Use is not recommended (has not been studied).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
Refer to adult dosing.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral:
Galafold: 123 mg [contains fd&c blue #2 (indigotine)]
No
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral:
Galafold: 123 mg [contains corn starch, fd&c blue #2 (indigotine)]
Oral: Administer every other day at the same time of day. Do not administer on 2 consecutive days. Administer on an empty stomach; do not consume food at least 2 hours before or 2 hours after the migalastat dose (for a minimum of 4 hours of fasting), although clear liquids may be consumed during the 4-hour fast. Swallow capsule whole; do not cut, crush, or chew.
Fabry disease: Treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
Migalastat may be confused with miglitol, miglustat.
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.
>10%:
Central nervous system: Headache (35%)
Gastrointestinal: Nausea (12%)
Genitourinary: Urinary tract infection (15%)
Respiratory: Nasopharyngitis (18%)
Miscellaneous: Fever (12%)
1% to 10%:
Gastrointestinal: Abdominal pain (9%), diarrhea (9%)
Neuromuscular & skeletal: Back pain (9%)
Respiratory: Cough (9%), epistaxis (9%)
There are no contraindications listed in the manufacturer's labeling.
Canadian labeling: Additional contraindications (not in US labeling): Hypersensitivity to migalastat or any component of the formulation or container
Disease-related concerns
• Renal impairment: Use is not recommended in patients with severe renal impairment (eGFR <30 mL/minute/1.73 m2) or with ESRD requiring dialysis.
Other warnings/precautions:
• Conversion: Migalastat 123 mg is equivalent to 150 mg of migalastat hydrochloride.
• Medication safety: Due to potential sound-alike/look-alike issues (migalastat may be confused with miglustat), use caution when selecting product for computerized order entry and for dispensing.
• Patient selection criteria: Select patients with confirmed Fabry disease for migalastat treatment if an amenable galactosidase alpha (GLA) variant is present. Migalastat is indicated for patients with an amenable GLA gene variant that is interpreted by a clinical geneticist as causing Fabry disease (pathogenic, likely pathogenic) in the patient's clinical context. Consultation with a clinical geneticist is strongly recommended in cases where clinical significance of the amenable GLA variant is uncertain or may be benign (not the cause of Fabry disease). For further information on amenable GLA variants, refer to the prescribing information or the manufacturer at 1-877-426-4287.
None known.
There are no known significant interactions.
Administration 1 hour before a high-fat meal (850 calories; 56% from fat) or a light meal (507 calories; 30% from fat) reduced the mean migalastat AUC by 37% to 42% and Cmax by 15% to 29%; Management: Administer 2 hours before or 2 hours after food.
Information related to use in pregnancy is limited.
Data collection to monitor pregnancy and infant outcomes following exposure to migalastat is ongoing. Health care providers are encouraged to enroll females exposed to migalastat during pregnancy in the Fabry Pregnancy Registry (1-888-239-0758).
It is not known if migalastat is present in breast milk.
According to the manufacturer, the decision to breastfeed during therapy should consider the risk of infant exposure, the benefits of breastfeeding to the infant, and benefits of treatment to the mother.
Data collection to monitor neonate and infant outcomes following exposure to migalastat via breast milk up to 1 year of age is ongoing. Health care providers are encouraged to enroll patients in the Fabry Pregnancy Registry (1-888-239-0758).
Determine presence of an amenable galactosidase alpha (GLA) gene variant (prior to therapy); monitor renal function (creatinine clearance).
Migalastat is an oral pharmacological chaperone that stabilizes certain mutant variants of alpha-galactosidase to increase enzyme trafficking to lysosomes (Germain 2016). Migalastat 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. Binding to the active site stabilizes alpha-Gal A allowing trafficking from the endoplasmic reticulum into the site of action, the lysosome.
Distribution: Vz/F: ~89 L (range: 77 to 133 L)
Protein binding: None detected
Metabolism: Dehydrogenated to 3 O-glucuronide conjugated minor metabolites (M1 to M3). UDPGT is a minor elimination pathway.
Bioavailability: ~75%; reduced if 1 hour before a high-fat meal (850 calories; 56% from fat) or 1 hour before or after a light meal (507 calories; 30% from fat)
Half-life elimination: ~4 hours
Time to peak: ~3 hours
Excretion: Urine (~77%; 80% as unchanged drug); feces (~20% as unchanged drug)
Altered kidney function: Systemic exposure is increased (significantly) in subjects with eGFR <30 mL/minute/1.73 m2
Capsules (Galafold Oral)
123 mg (per each): $2,362.71
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