Medically reviewed by Drugs.com. Last updated on Jan 3, 2019.
(mi GAL a stat)
- Migalastat Hydrochloride
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Galafold: 123 mg [contains fd&c blue #2 (indigotine)]
Brand Names: U.S.
- Pharmacologic Chaperone
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.
Vz/F: ~89 L (range: 77 to 133 L)
Dehydrogenated to 3 O-glucuronide conjugated minor metabolites (M1 to M3). UDPGT is a minor elimination pathway.
Urine (~77%; 80% as unchanged drug); feces (~20% as unchanged drug)
Time to Peak
Special Populations: Renal Function Impairment
Systemic exposure is increased (significantly) in subjects with eGFR <30 mL/minute/1.73 m2
Use: Labeled Indications
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.
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
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.
Refer to adult dosing.
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.
Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F).
There are no known significant interactions.
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%)
• Renal impairment: Use is not recommended in patients with severe renal impairment (eGFR <30 mL/minute/1.73 m2) or with ESRD requiring dialysis.
• 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.
Determine presence of an amenable galactosidase alpha (GLA) gene variant (prior to therapy); monitor renal function (creatinine clearance).
Information related to use in pregnancy is limited.
• Discuss specific use of drug and side effects with patient as it relates to treatment. (HCAHPS: During this hospital stay, were you given any medicine that you had not taken before? Before giving you any new medicine, how often did hospital staff tell you what the medicine was for? How often did hospital staff describe possible side effects in a way you could understand?)
• Patient may experience headache, rhinitis, pharyngitis, nausea, abdominal pain, back pain, cough, diarrhea, or epistaxis. Have patient report immediately to prescriber signs of a urinary tract infection (hematuria, burning or painful urination, polyuria, fever, lower abdominal pain, or pelvic pain) (HCAHPS).
• Educate patient about signs of a significant reaction (eg, wheezing; chest tightness; fever; itching; bad cough; blue skin color; seizures; or swelling of face, lips, tongue, or throat). Note: This is not a comprehensive list of all side effects. Patient should consult prescriber for additional questions.
Intended Use and Disclaimer: Should not be printed and given to patients. This information is intended to serve as a concise initial reference for health care professionals to use when discussing medications with a patient. You must ultimately rely on your own discretion, experience, and judgment in diagnosing, treating, and advising patients.
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
More about migalastat
- Side Effects
- During Pregnancy
- Dosage Information
- Drug Interactions
- En Español
- Drug class: miscellaneous metabolic agents
Other brands: Galafold