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Migalastat

Medically reviewed on Nov 15, 2018

Pronunciation

(mi GAL a stat)

Index Terms

  • AT1001
  • Galafold
  • Migalastat Hydrochloride

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Capsule, Oral:

Galafold: 123 mg [contains fd&c blue #2 (indigotine)]

Brand Names: U.S.

  • Galafold

Pharmacologic Category

  • Pharmacologic Chaperone

Pharmacology

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)

Metabolism

Dehydrogenated to 3 O-glucuronide conjugated minor metabolites (M1 to M3). UDPGT is a minor elimination pathway.

Excretion

Urine (~77%; 80% as unchanged drug); feces (~20% as unchanged drug)

Time to Peak

~3 hours

Half-Life Elimination

~4 hours

Protein Binding

None detected

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.

Contraindications

There are no contraindications listed in the manufacturer's labeling.

Dosing: Adult

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.

Dosing: Geriatric

Refer to adult dosing.

Administration

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.

Storage

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).

Drug Interactions

There are no known significant interactions.

Adverse Reactions

>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%)

Warnings/Precautions

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.

Monitoring Parameters

Determine presence of an amenable galactosidase alpha (GLA) gene variant (prior to therapy); monitor renal function (creatinine clearance).

Pregnancy Considerations

Information related to use in pregnancy is limited.

Patient Education

• 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.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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