migalastat

Contraindicated in:

Severe renal impairment or end-stage renal disease requiring dialysis.

Use Cautiously in:

OB: Safety not established in pregnancy;
Lactation: Safety not established in breastfeeding;
Pedi: Safety and effectiveness not established in children.

Adv. Reactions/Side Effects ⬆ ⬇

EENT: epistaxis, nasopharyngitis

GI: diarrhea, nausea, abdominal pain, vomiting

GU: urinary tract infection

MS: back pain

Neuro: headache

Resp: cough

Misc: fever

Interactions ⬆ ⬇

Drug-drug:

None reported.

Drug-Food:

Caffeine may ↓ levels and effectiveness; avoid caffeine ≥2 hr before and after administration of migalastat.

Availability ⬆ ⬇

Capsules: 123 mg

Route/Dosage ⬆ ⬇

PO (Adults ): 123 mg every other day.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Reversibly binds to the active site of the alpha-galactosidase A protein (encoded by GLA), which is deficient in Fabry disease. Binding to the active site stabilizes the alpha-galactosidase A protein, allowing trafficking from the endoplasmic reticulum into the site of action, the lysosome. In the lysosome, migalastat dissociates from alpha-Gal A, allowing it to break down the glycosphingolipids, globotriaosylceramide, and globotriaosylsphingosine.

Therapeutic effects:

Reduction in number of globotriaosylceramide inclusions per kidney interstitial capillary.

Classifications ⬆ ⬇

Therapeutic Classification: none assigned

Pharmacologic Classification: temporary class

Pharmacokinetics ⬆ ⬇

Absorption: 75% absorbed following oral administration; reduced if 1 hr before a high-fat meal or 1 hr before or after a light meal.

Distribution: Well distributed to tissues.

Metabolism/Excretion: Dehydrogenated to three O-glucuronide conjugated minor metabolites (M1–M3). Uridine diphosphate glucuronosyltransferase is a minor elimination pathway. Primarily excreted in the urine (77%; 62% as unchanged drug), with 20% excreted in the feces as unchanged drug.

Half-Life: 4 hr.

Time/Action Profile ⬆ ⬇

(plasma concentrations)

ROUTE	ONSET	PEAK	DURATION
PO	unknown	3 hr	unknown

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects of migalastat to patient. Tell patient to take once every other day at the same time of day and to avoid taking on 2 consecutive days. Take a missed dose only if it is within 12 hr of the normal time that the dose should have been taken. If >12 hr have passed, resume normal every-other-day dosing schedule at the next planned dosing day and time. Advise patient to read Patient Information before starting and with each Rx refill in case of changes.
Advise patient to take drug on an empty stomach and to not consume food or caffeine ≥2 hr before and 2 hr after taking drug to give a minimum 4-hr fast (water [plain, flavored, or sweetened], fruit juices without pulp, and caffeine-free carbonated beverages are allowable during this time).
Advise patient that migalastat may cause side effects of headache, nasal congestion, and fever.
Advise patient to report signs and symptoms of urinary tract infection (fever, chills, urinary pain or burning, urinary frequency, bloody urine, flank pain).
Advise patient of possible GI symptoms (diarrhea, nausea, vomiting, abdominal pain). Emphasize need for adequate hydration during fasting periods to prevent dehydration.
Advise patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Rep: Advise patient to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Administer during pregnancy only if needed. Patients are encouraged to enroll in the pregnancy exposure study or lactation study or to obtain more information by contacting the Pregnancy Coordinating Center at 1-888-239-0758, emailing [email protected], or visiting www.fabrypregnancyregistry.com.

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Indications ⬇

Contraind./Precautions ⬆ ⬇