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miglustat (Rx)Brand and Other Names:Zavesca

 
 
 

Dosing & Uses

AdultPediatricGeriatric

Dosage Forms & Strengths

capsule

  • 100mg
more...

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
more...

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

miglustat and

No Results

     
     activity indicator 
    No Interactions Found
    Interactions Found

    Contraindicated

      Serious - Use Alternative

        Significant - Monitor Closely

          Minor

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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 Category: X

            Lactation: Unknown; not recommended; discontinue drug or do not nurse

            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

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            Formulary

            FormularyPatient Discounts

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            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.
            6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
            NC NOT COVERED – Drugs that are not covered by the plan.
            Code Definition
            PA Prior Authorization
            Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
            QL Quantity Limits
            Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
            ST Step Therapy
            Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
            OR Other Restrictions
            Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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