Miglustat


Concise Prescribing Info
Indications/Uses
Listed in Dosage.
Dosage/Direction for Use
Adult : PO Gaucher Disease type 1 Initial: 100 mg tid. Dose may be reduced to 100 mg once daily-bid in cases of diarrhoea or tremor. Niemann-Pick type C disease Initial: 200 mg tid, reduce dose in case of diarrhoea.
Dosage Details
Oral
Gaucher disease type 1
Adult: Initially, 100 mg tid. Dose may be reduced to 100 mg once daily-bid in cases of diarrhoea or tremor.

Oral
Niemann-Pick type C disease
Adult: Initially, 200 mg tid, reduce dose in case of diarrhoea.
Child: ≤0.47 m2 BSA: 100 mg once daily; >0.47-0.73 m2 BSA: 100 mg bid; >0.73-0.88 m2 BSA: 100 mg tid; >0.88-1.25 m2 BSA: 200 mg bid; 12 years or >1.25 m2 BSA: Same as adult dose. Dose reduction may be necessary in case of diarrhoea.
Renal Impairment
Gaucher disease type 1
CrCl (mL/min) Dosage
<30 Not recommended.
30-50 100 mg once daily.
50-70 100 mg bid.

Niemann-Pick type C disease
CrCl (mL/min) Dosage
<30 Not recommended.
30-50 100 mg bid.
50-70 200 mg bid.
Administration
May be taken with or without food.
Contraindications
Pregnancy.
Special Precautions
Patient with history or significant gastrointestinal disease (e.g. inflammatory bowel diseases). Renal impairment. Children. Lactation.
Adverse Reactions
Significant: Peripheral neuropathy, tremor, diarrhoea, weight loss, reduced platelet count.
Blood and lymphatic system disorders: Thrombocytopenia.
Eye disorders: Visual disturbance.
Gastrointestinal disorders: Abdominal pain/distention, flatulence, nausea, vomiting, constipation, xerostomia, dyspepsia, bloating, epigastric pain.
General disorders and administration site conditions: Weakness, fatigue, malaise, asthenia, chills.
Metabolism and nutrition disorders: Anorexia.
Musculoskeletal and connective tissue disorders: Leg cramps, back pain, muscle spasm, heaviness in limbs.
Nervous system disorders: Headache, dizziness, memory impairment, ataxia, paraesthesia, hypoaesthesia, migraine, unsteady gait.
Psychiatric disorders: Depression, insomnia.
Reproductive system and breast disorders: Menstrual disease, decreased libido.
Patient Counseling Information
This drug may cause dizziness, if affected, do not drive or operate machinery.
MonitoringParameters
Assess neurological evaluation at baseline and every 6 months during therapy. Monitor renal function, weight, platelet count, nutritional status, frequency of bowel movement, and adverse events (e.g. tremor, peripheral neuropathy).
Food Interaction
Food may decrease rate of absorption.
Action
Description: Miglustat competitively and reversibly inhibits glucosylceramide synthase, an enzyme responsible for glycosylceramide and glycolipid synthesis, thereby reducing the rate of formation of these substrates which accumulate in Gaucher disease type 1 and Niemann-Pick type C disease respectively.
Pharmacokinetics:
Absorption: Rapidly absorbed. Bioavailability: 97%. Time to peak plasma concentration: 2-2.5 hours.
Distribution: Volume of distribution: 83-105 L.
Excretion: Mainly via urine (70-80%, as unchanged drug); faeces. Elimination half-life: 6-7 hours.
Chemical Structure

Click on icon to see table/diagram/image
Storage
Store between 20-25°C.
ATC Classification
A16AX06 - miglustat ; Belongs to the class of various alimentary tract and metabolism products.
Disclaimer: This information is independently developed by MIMS based on Miglustat from various references and is provided for your reference only. Therapeutic uses, prescribing information and product availability may vary between countries. Please refer to MIMS Product Monographs for specific and locally approved prescribing information. Although great effort has been made to ensure content accuracy, MIMS shall not be held responsible or liable for any claims or damages arising from the use or misuse of the information contained herein, its contents or omissions, or otherwise. Copyright © 2020 MIMS. All rights reserved. Powered by MIMS.com
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