Miglustat


Generic Medicine Info
Indications and Dosage
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

Chemical Structure Image
Miglustat

Source: National Center for Biotechnology Information. PubChem Database. Miglustat, CID=51634, https://pubchem.ncbi.nlm.nih.gov/compound/Miglustat (accessed on Jan. 22, 2020)

Storage
Store between 20-25°C.
ATC Classification
A16AX06 - miglustat ; Belongs to the class of various alimentary tract and metabolism products.
References
Anon. Miglustat. Lexicomp Online. Hudson, Ohio. Wolters Kluwer Clinical Drug Information, Inc. https://online.lexi.com. Accessed 15/12/2017.

Buckingham R (ed). Miglustat. Martindale: The Complete Drug Reference [online]. London. Pharmaceutical Press. https://www.medicinescomplete.com. Accessed 15/12/2017.

Joint Formulary Committee. Miglustat. British National Formulary [online]. London. BMJ Group and Pharmaceutical Press. https://www.medicinescomplete.com. Accessed 15/12/2017.

McEvoy GK, Snow EK, Miller J et al (eds). Miglustat. AHFS Drug Information (AHFS DI) [online]. American Society of Health-System Pharmacists (ASHP). https://www.medicinescomplete.com. Accessed 15/12/2017.

Zavesca Capsule (Actelion Pharmaceuticals US, Inc.). DailyMed. Source: U.S. National Library of Medicine. https://dailymed.nlm.nih.gov/dailymed/. Accessed 15/12/2017.

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