miglustat (Rx)

Brand and Other Names:Zavesca
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Dosing & Uses

AdultPediatricGeriatric

Dosage Forms & Strengths

capsule

  • 100mg

Gaucher Disease

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

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

Renal Impairment

Mild (CrCl 50-70 mL/min/1.73 m²): 100 mg PO q12hr

Moderate (CrCl 30-50 mL/min/1.73m²): 100 mg PO qDay

Severe (CrCl <30 mL/min/1.73m²): Not recommended

Dosage Forms & Strengths

capsule

  • 100mg

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

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Interactions

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)

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                  Adverse Effects

                  >10%

                  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%)

                  1-10%

                  Thrombocytopenia (6-10%)

                  Vomiting (4%)

                  Memory impairment (8%)

                  Migraine (6%)

                  Abdominal distension (8%)

                  Anorexia (7%)

                  Epigastric pain (<6%)

                  Thrombocytopenia (5-7%)

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                  Warnings

                  Contraindications

                  Hypersensitivity to product or components

                  Cautions

                  Cases of peripheral neuropathy have been reported; patients should undergo baseline & repeat neuro evaluation at 6 month intervals; 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

                  May affect spermatogenesis & sperm parameters; male patients should use reliable contraception

                  May increase clearance of Cerezyme (imiglucerase); concomitant use not recommended

                  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

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                  Pregnancy & Lactation

                  Pregnancy

                  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 six 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

                  Animal data

                  • 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

                  Lactation

                  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.

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                  Pharmacology

                  Mechanism of Action

                  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

                  Pharmacokinetics

                  Half-Life: 6-7 hr

                  Peak Plasma Time: 2-2.5 hr

                  Bioavailability: 97%

                  Protein Bound: Does not bind to plasma proteins

                  Vd: 83-105 L

                  Metabolism: Not metabolized

                  Excretion: Urine

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                  Images

                  BRAND FORM. UNIT PRICE PILL IMAGE
                  miglustat oral
                  -
                  100 mg capsule
                  miglustat oral
                  -
                  100 mg capsule
                  Zavesca oral
                  -
                  100 mg capsule

                  Copyright © 2010 First DataBank, Inc.

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                  Patient Handout

                  Patient Education
                  miglustat oral

                  MIGLUSTAT - ORAL

                  (mi-GLOO-stat)

                  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. 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, breast-feeding while using this drug is not recommended. Consult your doctor before breast-feeding.

                  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.Laboratory and/or medical tests (such as nerve function, kidney function, liver/spleen measurements, complete blood count including platelets) should be performed periodically to monitor your progress or check for side effects.

                  MISSED DOSE: If you miss a dose, take it as soon as you remember. If it is near the time of the next 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 March 2022. Copyright(c) 2022 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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                  Formulary

                  FormularyPatient Discounts

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                  The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

                  Tier Description
                  1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
                  2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
                  3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
                  4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                  5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
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                  NC NOT COVERED – Drugs that are not covered by the plan.
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                  Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.