Version May 2024
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, administer the missed dose 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.
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.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral:
Galafold: 123 mg [contains fd&c blue #2 (indigotine,indigo carmine)]
No
Capsules (Galafold Oral)
123 mg (per each): $2,562.00
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
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,indigo carmine)]
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 or caffeine at least 2 hours before or 2 hours after the migalastat dose (for a minimum of 4 hours of fasting), although water (plain, flavored, or sweetened), fruit juices without pulp, and caffeine-free carbonated beverages 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.
None known.
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the Lexicomp drug interactions program by clicking on the “Launch drug interactions program” link above.
Caffeine and Caffeine Containing Products: May decrease the serum concentration of MigALAstat. Risk X: Avoid combination
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.
Outcome data following maternal use of migalastat during pregnancy are limited.
Data collection to monitor pregnancy and infant outcomes following exposure to migalastat is ongoing. Health care providers are encouraged to enroll patients exposed to migalastat during pregnancy in the Fabry Pregnancy Registry (1-888-239-0758 or http://www.fabrypregnancyregistry.com).
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 or http://www.fabrypregnancyregistry.com).
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); a significant decrease in systemic exposure was seen with ~190 mg of caffeine at the time of dosing.
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
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