Version July 2025
Niemann-Pick disease type C: Limited data available:
Note: Therapy is recommended for patients with neurological, cognitive, or psychiatric disease manifestations (Ref). Infant data is very limited; reported infant neurological effects (aside from seizures) have included hypotonia, dysphagia, feeding difficulties (onset: 5 to 12 months of age); earliest reported miglustat therapy is 7 months of age (Ref). Doses may be initiated slowly and adjusted for tolerability, particularly GI effects (diarrhea which may require dietary and pharmacologic management) (Ref). Several months of therapy may be necessary to see clinical benefit (Ref).
Infants and Children <12 years: Zavesca (and generics):
BSA ≤0.47 m2: Oral: 100 mg once daily.
BSA >0.47 to 0.73 m2: Oral: 100 mg twice daily.
BSA >0.73 to 0.88 m2: Oral: 100 mg 3 times daily.
BSA >0.88 to 1.25 m2: Oral: 200 mg twice daily.
BSA >1.25 m2: Oral: 200 mg 3 times daily.
Children ≥12 years and Adolescents: Zavesca (and generics): Oral: 200 mg 3 times daily (Ref).
Niemann-Pick Type C disease (Ref):
Children <12 years:
CrCl 50 to 70 mL/minute/1.73 m2: Administer two-thirds of regular BSA adjusted dose in 2 equal doses.
CrCl 30 to 50 mL/minute/1.73 m2: Administer one-third of regular BSA adjusted dose in 2 equal doses.
CrCl <30 mL/minute/1.73 m2: Not recommended.
Children ≥12 years and Adolescents:
CrCl 50 to 70 mL/minute/1.73 m2: 200 mg twice daily.
CrCl 30 to 50 mL/minute/1.73 m2: 100 mg twice daily.
CrCl <30 mL/minute/1.73 m2: Not recommended.
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied). However, dosage adjustment unlikely because miglustat is not metabolized by the liver.
(For additional information see "Miglustat: Drug information")
Gaucher disease, type 1: Zavesca (and generics): Oral: 100 mg 3 times daily; dose may be reduced to 100 mg 1 to 2 times daily in patients with adverse effects (eg, tremor, diarrhea).
Niemann-Pick disease type C (off-label use): Oral: 200 mg 3 times daily (Ref).
Pompe disease (lysosomal acid alpha-glucosidase deficiency), late onset:
Note: For use in combination with cipaglucosidase alfa; initiate 2 weeks after last enzyme replacement therapy dose. Dose is based on actual body weight.
Opfolda:
Weight ≥40 to <50 kg: Oral: 195 mg every other week, 1 hour before cipaglucosidase alfa dose.
Weight ≥50 kg: Oral: 260 mg every other week, 1 hour before cipaglucosidase alfa dose.
Missed doses:
If miglustat dose is missed: Do not administer cipaglucosidase alfa; reschedule next miglustat dose at least 24 hours after last miglustat dose.
If both miglustat and cipaglucosidase alfa doses are missed: Restart treatment as soon as possible.
Gaucher disease, type 1:
Zavesca (and generics):
CrCl >70 mL/minute/1.73 m2: No dosage adjustment necessary.
CrCl 50 to 70 mL/minute/1.73 m2: 100 mg twice daily.
CrCl 30 to 50 mL/minute/1.73 m2: 100 mg once daily.
CrCl <30 mL/minute/1.73 m2: Use is not recommended.
Niemann-Pick type C disease (off-label use) (Ref):
CrCl >70 mL/minute/1.73 m2: No dosage adjustment necessary.
CrCl 50 to 70 mL/minute/1.73 m2: 200 mg twice daily.
CrCl 30 to 50 mL/minute/1.73 m2: 100 mg twice daily.
CrCl <30 mL/minute/1.73 m2: Use is not recommended.
Pompe disease (lysosomal acid alpha-glucosidase deficiency), late-onset:
Opfolda:
CrCl ≥60 mL/minute: No dosage adjustment necessary.
CrCl 15 to 59 mL/minute:
Weight ≥40 to <50 kg: 130 mg every other week.
Weight ≥50 kg: 195 mg every other week.
CrCl <15 mL/minute: Use is not recommended (has not been studied).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied). However, dosage adjustment unlikely because miglustat is not metabolized by the liver.
The following adverse drug reactions are derived from product labeling unless otherwise specified. Adverse reactions reported in adults.
Zavesca administered for type 1 Gaucher disease:
>10%:
Endocrine & metabolic: Weight loss
Gastrointestinal: Abdominal pain, diarrhea, flatulence, nausea, vomiting
Nervous system: Asthenia, dizziness, headache, tremor
Neuromuscular & skeletal: Lower limb cramp, muscle cramps
Ophthalmic: Visual disturbance
1% to 10%:
Endocrine & metabolic: Menstrual disease
Gastrointestinal: Abdominal distention, anorexia, bloating, constipation, dyspepsia, epigastric pain, xerostomia
Hematologic & oncologic: Thrombocytopenia
Nervous system: Feeling of heaviness (limbs), memory impairment, migraine, paresthesia, peripheral neuropathy, unsteady gait
Neuromuscular & skeletal: Back pain
Opfolda administered in combination with Pombiliti for late-onset Pompe disease:
1% to 10%:
Cardiovascular: Flushing (2%), tachycardia (2%; including sinus tachycardia)
Dermatologic: Pruritus (2%), skin rash (4%), urticaria (2%)
Gastrointestinal: Abdominal distention (4%), abdominal pain (2%), diarrhea (6%), dysgeusia (2%), nausea (2%)
Nervous system: Chills (2%), dizziness (5%), headache (8%)
Neuromuscular & skeletal: Muscle spasm (2%)
Respiratory: Dyspnea (4%)
Miscellaneous: Fever (4%)
Frequency not defined:
Cardiovascular: Increased blood pressure
Gastrointestinal: Constipation, dyspepsia
Hematologic & oncologic: Decreased platelet count
Local: Infusion-site reaction (swelling)
Nervous system: Asthenia, drowsiness, fatigue, malaise, pain, paresthesia, tremor
Neuromuscular & skeletal: Arthralgia, myalgia
Renal: Flank pain
Postmarketing (any indication): Nervous system: Psychosis
Pregnancy (Opfolda only).
Canadian labeling: Hypersensitivity to miglustat or any component of the formulation; females who are or may become pregnant.
Concerns related to adverse effects:
• Diarrhea: Observed in the majority of patients, many also reported weight loss (within first 12 months of treatment). Diarrhea decreased over time with continued treatment, and may respond to diet modification (eg, reduction of sucrose, lactose and other carbohydrate intake), taking miglustat between meals, and/or to anti-diarrheal medications. If diarrhea occurs during treatment, instruct patients to avoid foods with high carbohydrate content. If symptoms persist, evaluate patients for underlying GI disease.
• Peripheral neuropathy: Peripheral neuropathy has been reported; neurologic monitoring is required. Weigh risk versus benefit of therapy if patient develops symptoms (eg, numbness and tingling); treatment discontinuation may be necessary.
• Platelet counts decreased: Mild decrease in platelet counts (without bleeding) has been observed in patients with type 1 Gaucher disease switched from enzyme replacement therapy; monitor platelet counts during therapy.
• Tremor: New-onset or exacerbations of existing tremor may occur. Tremor typically begins within the first month of treatment and may resolve over time (1 to 3 months) or respond to dosage reduction. Treatment discontinuation may be necessary if tremor does not respond within days of dose reduction.
Other warnings/precautions:
• Experienced physician: Should be administered under the supervision of a physician experienced in treatment of Gaucher disease.
• Registry: A registry has been established and all patients with Gaucher disease, and physicians who treat Gaucher disease are encouraged to participate. Information on the International Collaborative Gaucher Group (ICGG) Gaucher Registry may be obtained at https://www.registrynxt.com or by calling 1-888-404-4413.
Growth reductions, both weight and height, have been reported in pediatric patients with Niemann-Pick type C disease on miglustat therapy; initially with therapy, a weight loss was observed that may be accompanied with or followed by a decrease in height velocity. Monitor height and weight with therapy, assess risk/benefit periodically of miglustat therapy (Zavesca prescribing information [European Medicines Agency 2022]).
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral:
Opfolda: 65 mg [contains corn starch]
Yargesa: 100 mg
Zavesca: 100 mg
Generic: 100 mg
Yes
Capsules (migLUstat Oral)
100 mg (per each): $321.48
Capsules (Opfolda Oral)
65 mg (per each): $40.22
Capsules (Yargesa Oral)
100 mg (per each): $321.48
Capsules (Zavesca Oral)
100 mg (per each): $225.29
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Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral:
Zavesca: 100 mg [contains soybean lecithin]
Generic: 100 mg
Oral: Zavesca (and generics): May be administered with or without food; administration between meals may decrease the incidence of diarrhea. Doses should be taken at the same time each day at regular intervals. If patient is unable to tolerate or swallow capsule whole and powder is administered, mix powder into a liquid immediately prior to use (do not store); sweetening agents are not expected to interact (data on file [Actelion pharmaceuticals] 2011).
Oral:
Opfolda: Swallow capsules whole only with unsweetened beverages; do not consume other beverages or food for at least 2 hours before and at least 2 hours after administration. Administer 1 hour before IV cipaglucosidase alfa.
Zavesca (and generics): May be administered with or without food; administration between meals may decrease the incidence of diarrhea. Capsules should be taken at the same time each day at regular intervals. If patient is unable to tolerate or swallow capsule whole and powder is administered, mix powder into a liquid immediately prior to use (do not store); sweetening agents are not expected to interact (data on file [Actelion Pharmaceuticals Ltd 2011])
Opfolda: Store in original container at 20°C to 25°C (68°F to 77°F). Brief exposure to 15°C to 30°C (59°F to 86°F) permitted. Protect from light.
Zavesca (and generics): Store at 20°C to 25°C (68°F to 77°F). Brief exposure to 15°C to 30°C (59°F to 86°F) permitted. Note: Extended storage information may be available; contact product manufacturer to obtain current recommendations.
Treatment of mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not a therapeutic option (eg, due to allergy, hypersensitivity, or poor venous access) (FDA approved in adults); treatment of late-onset Pompe disease (lysosomal acid alpha-glucosidase deficiency), in combination with cipaglucosidase alfa, in patients who are not improving on enzyme replacement therapy (Opfolda: FDA approved in adults weighing ≥40 kg); has also been used to treat Niemann-Pick Type C Disease.
MigLUstat may be confused with migALAstat, miglitol.
Opfolda may be confused with Opdivo.
The Institute for Safe Medication Practices (ISMP) includes this medication among its list of drugs (contraindicated in pregnancy) which have a heightened risk of causing significant patient harm when used in error (High-Alert Medications in Community/Ambulatory Care Settings).
Zavesca: Brand name for miglustat [US, Canada, and multiple international markets], but also brand name for escitalopram [in multiple international markets; ISMP April 21, 2010]
Substrate of OCT1, OCT2, P-glycoprotein (Minor); Note: Assignment of Major/Minor substrate status based on clinically relevant drug interaction potential;
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 drug interactions program
There are no known significant interactions.
Patients with diarrhea should avoid foods with high carbohydrate content.
When miglustat is used in combination with cipaglucosidase alfa for Pompe disease (lysosomal acid alpha-glucosidase deficiency), evaluate pregnancy status prior to use; verify the patient is not pregnant prior to treatment initiation. Patients who may become pregnant should use effective contraception during therapy with cipaglucosidase alfa and for at least 60 days after the last dose.
Based on a 6-week study of 7 healthy patients, miglustat is not expected to decrease male fertility.
Based on data from animal reproduction studies, in utero exposure to miglustat may cause fetal harm.
Uncontrolled type 1 Gaucher disease is associated an increased risk of spontaneous abortion; maternal hepatosplenomegaly and thrombocytopenia may also occur and lead to adverse pregnancy outcomes.
When used in combination with cipaglucosidase alfa for Pompe disease (lysosomal acid alpha-glucosidase deficiency), miglustat is contraindicated during pregnancy.
Neurologic evaluations baseline and repeated every 6 months; platelet count; renal function; growth parameters in pediatric patients (height, weight, head circumference) (Patterson 2012)
Miglustat competitively and reversibly inhibits the enzyme needed to produce glycosphingolipids and decreases the rate of glycosphingolipid glucosylceramide formation. Glucosylceramide accumulates in type 1 Gaucher disease, causing complications specific to this disease. In patients receiving cipaglucosidase alfa, miglustat reduces endogenous inactivation of cipaglucosidase alfa-atga in the blood.
Distribution: Vd: Opfolda: ~94 L; Zavesca: 83 to 105 L.
Protein binding: No binding to plasma proteins.
Metabolism: No evidence of metabolism in humans.
Bioavailability: 97%.
Half-life elimination: Opfolda: ~6 hours; Zavesca: 6 to 7 hours.
Time to peak, plasma: Opfolda: 2 to 3 hours; Zavesca: 2 to 2.5 hours.
Excretion: Urine (as unchanged drug).
Clearance: Opfolda: 10 L/hour.
Altered kidney function: Limited data suggests that the clearance of miglustat decreases 40% and 60% with mild and moderate renal impairment, respectively. A decreased clearance of 70% has been suggested in patients with severe renal impairment.
