miglustat (Rx)

Brand and Other Names:Zavesca, Opfolda

Dosing & Uses


Dosage Forms & Strengths


  • 65mg (Opfolda)
  • 100mg (Zavesca)

Gaucher Disease

Zavesca only

Indicated as monotherapy for treatment of adults with mild-to-moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option

100 mg PO q8hr; decrease to qDay or q12hr for patients with adverse effects (eg, tremor, diarrhea)

Pompe Disease

Opfolda only

Indicated in combination with cipaglucosidase alfa, a hydrolytic lysosomal glycogen-specific enzyme, for adults with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg who are not improving on their current enzyme replacement therapy (ERT)

Refer to cipaglucosidase alfa information for dosage and administration recommendations

Dose based on actual body weight

  • 40 kg to <50 kg: 195 mg PO every other week
  • ≥50 kg: 260 mg PO every other week
  • Start miglustat combination with cipaglucosidase alfa 2 weeks after last ERT dose
  • Take miglustat 1 hr before administering cipaglucosidase alfa IV

Dosage Modifications

Renal impairment (Zavesca)

  • Mild (CrCl 50-70 mL/min/1.73 m²): 100 mg PO q12hr
  • Moderate (CrCl 30-50 mL/min/1.73 m²): 100 mg PO qDay
  • Severe (CrCl <30 mL/min/1.73 m²): Not recommended

Renal impairment (Opfolda)

  • Mild (CrCl 60-89 mL/min): No dosage adjustment necessary
  • Moderate-to-severe (CrCl 15-59 mL/min)
    • 40 kg to <50 kg: 130 mg PO every other week
    • ≥50 kg: 195 mg PO every other week

Dosing Considerations

Opfolda: Verify pregnancy status of females of reproductive potential before initiating

Neimann-Pick Disease (Orphan)

Treatment of the neurological manifestations of Niemann-Pick disease type C

Orphan indication sponsor

  • Amicus Therapeutics, Inc; 6 Cedar Brook Drive; Cranbury, NJ 08512

Dosage Forms & Strengths


  • 100mg (Zavesca)

Gaucher Disease

Safety and efficacy not established

Niemann-Pick Type C Disease (Off-Label)

Approved in Europe for NP-C in children >4 years

Limited data suggests a dose of 200 mg PO q8hr; adjust for body surface area with young children as follows

BSA >1.25 m²: 200 mg PO q8hr

BSA >0.88 - 1.25 m²: 200 mg PO q12hr

BSA >0.73-0.88 m²: 100 mg PO q8hr

BSA >0.47-0.73 m²: 100 mg PO q12hr

BSA ≤0.47 m²: 100 mg PO qDay

Monitoring: Reduced growth may occur in children because of diarrhea and weight loss; monitor during treatment

For elderly, dose with caution at low end of dosing range



Interaction Checker

and miglustat

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                • imiglucerase

                  miglustat decreases levels of imiglucerase by increasing renal clearance. Use Caution/Monitor. Effects on imiglucerase levels variable; concomitant use not recommended by mfr of miglustat (Zavesca).

                Minor (0)


                  Adverse Effects



                  • Diarrhea (89-100%)
                  • Weight loss (66-70%)
                  • Abdominal pain (18-67%)
                  • Flatulence (29-50%)
                  • Dizziness (11%),
                  • Headache (21-22%)
                  • Tremor (11-30%)
                  • Nausea (8-22%)
                  • Vomiting (4-11%)
                  • Visual disturbances (17%)



                  • Thrombocytopenia (6-10%)
                  • Vomiting (4%)
                  • Memory impairment (8%)
                  • Migraine (6%)
                  • Abdominal distension (8%)
                  • Anorexia (7%)
                  • Epigastric pain (<6%)
                  • Thrombocytopenia (5-7%)

                  Opfolda with cipaglucosidase alfa

                  • Headache (8.2%)
                  • Diarrhea (5.9%)
                  • Dizziness (4.7%)
                  • Dyspnea (3.5%)
                  • Abdominal distention (3.5%)
                  • Pyrexia (3.5%)
                  • Rash (3.5%)
                  • Abdominal pain (2.4%)
                  • Nausea (2.4%)
                  • Chills (2.4%)
                  • Dysgeusia (2.4%)
                  • Flushing (2.4%)
                  • Muscle spasms (2.4%)
                  • Pruritus (2.4%)
                  • Tachycardia (2.4%)
                  • Urticaria (2.4%)
                  • ≥2%
                    • Myalgia
                    • Arthralgia
                    • Increased blood pressure
                    • Pain
                    • Tremor
                    • Dyspepsia
                    • Asthenia
                    • Constipation
                    • Infusion site swelling
                    • Flank pain
                    • Malaise
                    • Paresthesia
                    • Decreased platelet count



                  Hypersensitivity to product or components

                  Pregnancy (Opfolda in combination with cipaglucosidase alfa)


                  Cases of peripheral neuropathy reported; baseline and repeat neurologic evaluation at 6 month intervals recommended; consider treatment cessation if symptoms develop

                  Tremor may develop or be exacerbated; dose reduction may ameliorate, usually within days; discontinuation may be required

                  Avoid high carbohydrate foods during treatment to lessen side effect of diarrhea; weight loss reported due to the diarrhea; incidence decreases over time

                  MIld decrease in platelet counts reported; bleeding not reported

                  Exacerbation of existing tremor may occur that may resolve over time or improve with dose reduction; may consider discontinuing therapy if it does not resolve


                  • Opfolda: May cause embryo-fetal harm when administered to pregnant females and is contraindicated during pregnancy; refer to cipaglucosidase alfa prescribing information for a description of additional risks and warnings with its administration
                  • May affect spermatogenesis and sperm parameters; male patients should use reliable contraception

                  Drug interaction overview

                  • May increase clearance of imiglucerase by 70%; these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase; caution with concomitant use

                  Pregnancy & Lactation


                  Based on findings from animal reproduction studies, drug may cause fetal harm when administered to a pregnant woman; available data from postmarketing case reports with drug use in pregnancy are insufficient to assess a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes; there are risks associated with symptomatic Type I Gaucher disease in pregnancy, including hepatosplenomegaly and thrombocytopenia; advise pregnant women of potential risks to the fetus

                  Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations; Type 1 Gaucher disease manifestations may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of a pregnancy and thrombocytopenia which can lead to increased bleeding and possible hemorrhage

                  Reproductive potential

                  • Findings from a small clinical study in seven healthy adult males who received therapy for 6 weeks did not indicate effects on male fertility; studies in male rats have shown that drug decreased fertility but findings were reversible; studies in female rats have shown increased post-implantation loss and decreased embryo-fetal survival
                  • When used in combination with cipaglucoasidase alfa for Pompe disease may impair human female fertility


                  • Advise females of reproductive potential to use effective contraception during treatment with miglustate in combination with cipaglucoasidase alfa and for at least 60 days after the last dose
                  • May affect spermatogenesis and sperm parameters; male patients should use reliable contraception

                  Animal data

                  • Opfolda
                    • In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with oral miglustat in combination with cipaglucosidase alfa at 3-fold and 16-fold, respectively, the maximum recommended human dose (MRHD) based on plasma AUC exposure
                  • Zavesca
                    • In animal reproduction studies, drug was maternally toxic in rabbits at exposures near the expected human therapeutic dose and caused embryo-fetal toxicities in rats at doses twice recommended human dose; no adverse developmental outcomes were observed with administration of drug to pregnant rats at dose levels 6 times recommended human dose


                  There are no available data on presence of drug in either human or animal milk, effects on breastfed infant, or on milk production; based on physical properties of miglustat, drug is likely to be present in breast milk; because of potential for serious adverse reactions in breastfed infants, advise women that breastfeeding is not recommended

                  Pregnancy Categories

                  A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

                  B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

                  C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

                  D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

                  X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

                  NA: Information not available.



                  Mechanism of Action

                  Type 1 Gaucher disease (Zevesca)

                  • Type 1 Gaucher disease is caused by a functional deficiency of glucocerebrosidase, the enzyme that mediates glycosphingolipid glucosylceramide degradation
                  • Miglustat is a competitive and reversible inhibitor of glucosylceramide synthase, the initial enzyme in a series of reactions resulting in synthesis of most glygosphingolipids; decreases rate of glycosphingolipid glucosylceramide formation

                  Pompe disease (Opfolda)

                  • Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa in the blood after infusion
                  • Bound miglustat is dissociated from cipaglucosidase alfa after it is internalized and transported into lysosomes
                  • Miglustat alone has no pharmacological activity in cleaving glycogen



                  • Peak plasma time: 2-2.5 hr
                  • Steady-state achieved: 1.5-2 days
                  • Food: Peak plasma levels decreased by 36% and time to peak delayed 2 hr; but no statistically significant effect on extent of absorption


                  • Peak plasma time: 2-3 hr
                  • Peak plasma concentration: 3 mcg/mL
                  • AUC: 25 mcg⋅hr/mL
                  • Food: Predicted to result in delayed absorption and decreased peak plasma concentation


                  Protein bound: Does not bind to plasma proteins


                  • Vd: 83-105 L
                  • At steady-state, concentration in CSF wqas 31.4-67.2% of plasma


                  • Vd: 94 L


                  Major route of excretion of miglustat is via kidney



                  • Half-life: 6-7 hr
                  • Excretion: Urine 83%; feces 12%


                  • Half-life: 6 hr
                  • Clearance: 10 L/hr



                  Oral Administration


                  • May take with or without food


                  • Start in combination with cipaglucosidase alfa 2 weeks after last ERT dose
                  • Take miglustat ~1 hr before IV administration of cipaglucosidase alfa
                  • Swallow miglustat capsules whole only with unsweetened beverages (eg, water, tea or coffee with no cream, sugar, or sweeteners)
                  • Do not consume other beverages or food for at least 2 hr before before and 2 hr after administration
                  • Missed dose
                    • If missed miglustat only, do not administer cipaglucosidase alfa and reschedule treatment at least 24 hr after miglustat was last taken
                    • If miglustat in combination with cipaglucosidase alfa are both missed, restart treatment as soon as possible


                  Store at 20-25ºC (68-77ºF); excursions permitted between 15-30ºC (59-86ºF)

                  Store in original container to protect from light



                  miglustat oral
                  100 mg capsule
                  miglustat oral
                  100 mg capsule
                  Zavesca oral
                  100 mg capsule
                  Opfolda oral
                  65 mg capsule

                  Copyright © 2010 First DataBank, Inc.


                  Patient Handout

                  Patient Education
                  miglustat oral

                  MIGLUSTAT - ORAL


                  COMMON BRAND NAME(S): Zavesca

                  USES: Miglustat is used for the long-term treatment of a certain rare genetic problem (Gaucher's disease). It works by decreasing the amount of a certain natural fatty substance (glucosylceramide) that builds up due to this condition. This can help improve symptoms of Gaucher's disease, such as tiredness, pain in the joints/bones, and a swollen liver or spleen.

                  HOW TO USE: Read the Patient Information Leaflet if available from your pharmacist before you start taking miglustat and each time you get a refill. If you have any questions, ask your doctor or pharmacist.Take this medication by mouth with or without food as directed by your doctor, usually 3 times a day. The dosage is based on your medical condition and response to treatment.Take this medication regularly to get the most benefit from it. To help you remember, take it at the same times each day.Tell your doctor if your condition does not improve or if it worsens.

                  SIDE EFFECTS: Nausea, vomiting, stomach/abdominal pain, gas, diarrhea, weight loss, headache, muscle cramps, dizziness, or weakness may occur. If any of these effects last or get worse, tell your doctor or pharmacist promptly.To reduce diarrhea, your doctor may tell you to take miglustat between meals, recommend that you change your diet to avoid high-carbohydrate foods, or prescribe a drug (such as loperamide) to treat diarrhea. Talk to your doctor or pharmacist for more details.Remember that this medication has been prescribed because your doctor has judged that the benefit to you is greater than the risk of side effects. Many people using this medication do not have serious side effects.Tell your doctor right away if you have any serious side effects, including: numbness/tingling/weakness/pain of the hands or feet, new/worsening shaking or tremor of the hands, vision changes, new/worsening bruising/bleeding.A very serious allergic reaction to this drug is rare. However, get medical help right away if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.This is not a complete list of possible side effects. If you notice other effects not listed above, contact your doctor or pharmacist.In the US -Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.

                  PRECAUTIONS: Before taking miglustat, tell your doctor or pharmacist if you are allergic to it; or if you have any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.Before using this medication, tell your doctor or pharmacist your medical history, especially of: kidney disease, numbness/tingling of the hands/feet, stomach/intestinal problems (such as inflammatory bowel disease).This drug may make you dizzy. Alcohol or marijuana (cannabis) can make you more dizzy. Do not drive, use machinery, or do anything that needs alertness until you can do it safely. Limit alcoholic beverages. Talk to your doctor if you are using marijuana (cannabis).Before having surgery, tell your doctor or dentist about all the products you use (including prescription drugs, nonprescription drugs, and herbal products).During pregnancy, this medication should be used only when clearly needed. It may harm an unborn baby. Discuss the risks and benefits with your doctor.It is unknown if this medication passes into breast milk. Because of the possible risk to the infant, breastfeeding is not recommended while using this medication. Consult your doctor before breastfeeding.

                  DRUG INTERACTIONS: Drug interactions may change how your medications work or increase your risk for serious side effects. This document does not contain all possible drug interactions. Keep a list of all the products you use (including prescription/nonprescription drugs and herbal products) and share it with your doctor and pharmacist. Do not start, stop, or change the dosage of any medicines without your doctor's approval.

                  OVERDOSE: If someone has overdosed and has serious symptoms such as passing out or trouble breathing, call 911. Otherwise, call a poison control center right away. US residents can call their local poison control center at 1-800-222-1222. Canada residents can call a provincial poison control center.

                  NOTES: Do not share this medication with others.Lab and/or medical tests (such as nerve function, kidney function, liver/spleen measurements, complete blood count including platelets) should be done while you are taking this medication. Keep all medical and lab appointments. Consult your doctor for more details.

                  MISSED DOSE: If you miss a dose, skip the missed dose. Take your next dose at the regular time. Do not double the dose to catch up.

                  STORAGE: Store at room temperature away from light and moisture. Do not store in the bathroom. Keep all medications away from children and pets.Do not flush medications down the toilet or pour them into a drain unless instructed to do so. Properly discard this product when it is expired or no longer needed. Consult your pharmacist or local waste disposal company.

                  Information last revised October 2023. Copyright(c) 2023 First Databank, Inc.

                  IMPORTANT: HOW TO USE THIS INFORMATION: This is a summary and does NOT have all possible information about this product. This information does not assure that this product is safe, effective, or appropriate for you. This information is not individual medical advice and does not substitute for the advice of your health care professional. Always ask your health care professional for complete information about this product and your specific health needs.



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