miglustat

REMS

Zavesca

Mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not an option.

Opfolda

Late-onset Pompe disease in patients who are not improving on their current enzyme replacement therapy (in combination with cipaglucosidase alfa).

Contraind./Precautions ⬆ ⬇

Contraindicated in:

Hypersensitivity;
Severe renal impairment (<30 mL/min);
OB: Pregnancy (Opfolda);
Lactation: Lactation.

Use Cautiously in:

Mild to moderate renal impairment (dose alteration recommended if CCr <70 mL/min [Zavesca] or <60 mL/min [Opfolda]);
Rep: Women of reproductive potential (Opfolda);
OB: Safety not established in pregnancy (Zavesca);
Pedi: Safety and effectiveness not established in children;
Geri: Consider age-related ↓ in body mass and cardiac, renal and hepatic function; other chronic illnesses; and concurrent drug therapies in older adults.

Adv. Reactions/Side Effects ⬆ ⬇

CV: ↑BP, tachycardia

Derm: flushing, pruritus, rash, urticaria

GI: abdominal pain, diarrhea, flatulence, nausea, abdominal distention, abdominal pain, anorexia, constipation, dyspepsia

GU: ↓fertility

Hemat: thrombocytopenia

Metab: weight loss

MS: arthralgia, muscle spasm, myalgia

Neuro: headache, dizziness, dysgeusia, paresthesia, peripheral neuropathy, sedation, tremor

Misc: chills, fever

Interactions ⬆ ⬇

Drug-drug:

↓levels and effectiveness of imiglucerase; avoid concurrent use.

Availability ⬆ ⬇

(Generic available)

Capsules (Zavesca): 100 mg
Capsules (Opfolda): 65 mg

Route/Dosage ⬆ ⬇

Zavesca

PO (Adults ): 100 mg 3 times daily at regular intervals.

Renal Impairment

PO (Adults ): CCr 50–70 mL/min— 100 mg twice daily; CCr 30–50 mL/min— 100 mg once daily.

Opfolda

PO (Adults ≥50 kg): 260 mg every other wk (given 1 hr before administration of cipaglucosidase). To be started in combination with cipaglucosidase 2 wk after last enzyme replacement therapy dose.
PO (Adults 40–<50 kg): 195 mg every other wk (given 1 hr before administration of cipaglucosidase). To be started in combination with cipaglucosidase 2 wk after last enzyme replacement therapy dose.

Renal Impairment

PO (Adults ≥50 kg): CCr 15–59 mL/min—195 mg every other wk (given 1 hr before administration of cipaglucosidase). To be started in combination with cipaglucosidase 2 wk after last enzyme replacement therapy dose.

Renal Impairment

PO (Adults 40–<50 kg): CCr 15–59 mL/min—130 mg every other wk (given 1 hr before administration of cipaglucosidase). To be started in combination with cipaglucosidase 2 wk after last enzyme replacement therapy dose.

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Competitively and reversibly inhibits glucosylceramide synthase, which is the initial step in the production of glycosphingolipids. The glycosphingolipid glucosylceramide accumulates in tissue in Gaucher disease. In Pompe disease, it stabilizes and reduces inactivation of cipaglucosidase alfa in the blood after its infusion.

Therapeutic effects:

Decreased production/accumulation of glycosphingolipid glucosylceramide with decreased tissue damage.
Improved efficacy of cipaglucosidase alfa.

Classifications ⬆ ⬇

Therapeutic Classification: none assigned

Pharmacologic Classification: enzyme inhibitors (D-glucose analogue), substrate reduction therapy

Pharmacokinetics ⬆ ⬇

Absorption: Well absorbed following oral administration.

Distribution: Distributes into extravascular tissues.

Metabolism/Excretion: Not metabolized; excreted mostly unchanged in urine.

Half-Life: 6–7 hr.

Time/Action Profile ⬆ ⬇

(plasma concentrations)

ROUTE	ONSET	PEAK	DURATION
PO	unknown	2–3 hr	8 hr

Patient/Family Teaching ⬆ ⬇

Explain purpose and side effects of medication to patient. Advise patient to read Patient Information before starting therapy. If a dose is missed, skip the dose and take at the next usual time. Advise patient that sharing of this medication may be dangerous.
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.
Advise patient to notify health care professional if frequent diarrhea occurs. May be prescribed an antidiarrheal medication.
Instruct patient to notify health care professional if numbness, pain, or burning in the hands and feet occur or if tremor develops or existing tremor worsens.
Rep: May cause fetal harm. Advise females of reproductive potential to notify health care professional if pregnancy is planned or suspected and to avoid breastfeeding during therapy.

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Indications ⬇

Contraind./Precautions ⬆ ⬇

Adv. Reactions/Side Effects ⬆ ⬇

Interactions ⬆ ⬇

Availability ⬆ ⬇

Route/Dosage ⬆ ⬇

US Brand Names ⬆ ⬇

Action ⬆ ⬇

Classifications ⬆ ⬇

Pharmacokinetics ⬆ ⬇

Time/Action Profile ⬆ ⬇

Patient/Family Teaching ⬆ ⬇

Pronunciation ⬆