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.
Gaucher disease type 1
100 mg once daily.
100 mg bid.
Niemann-Pick type C disease
200 mg bid.
May be taken with or without food.
Patient with history or significant gastrointestinal disease (e.g. inflammatory bowel diseases). Renal impairment. Children. Lactation.
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.
This drug may cause dizziness, if affected, do not drive or operate machinery.
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 may decrease rate of absorption.
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.
A16AX06 - miglustat ; Belongs to the class of various alimentary tract and metabolism products.
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