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Miglustat Gen.Orph is a brand name for Miglustat. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable (see sections 4.4 and 5.1). Miglustat Gen.Orph is indicated for the treatment of…
Verbatim from this product's EMA label. Tap a section to expand.
Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate. Posology Dose in type 1 Gaucher disease Adult The recommended starting dose for the treatment of adult patients with type 1 Gaucher disease is 100 mg three times a day.
Temporary dose reduction to 100 mg once or twice a day may be necessary in some patients because of diarrhoea. Paediatric population The efficacy of miglustat in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established.
No data are available. Dose in Niemann-Pick type C disease Adult The recommended dose for the treatment of adult patients with Niemann-Pick type C disease is 200 mg three times a day. 3 Paediatric population The recommended dose for the treatment of adolescent patients (12 years of age and above) with Niemann-Pick type C disease is 200 mg three times a day.
Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below: Table 1. 47 100 mg once a day Temporary dose reduction may be necessary in some patients because of diarrhoea. 4).
There is limited experience with the use of miglustat in Niemann-Pick type C disease patients under the age of 4 years. Special populations Elderly There is no experience with the use of miglustat in patients over the age of 70. Renal impairment Pharmacokinetic data indicate increased systemic exposure to miglustat in patients with renal impairment.
73 m2, administration should commence at a dose of 100 mg twice daily in patients with type 1 Gaucher disease and at a dose of 200 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
73 m2, administration should commence at a dose of 100 mg once daily in patients with type 1 Gaucher disease and at a dose of 100 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.
2). Hepatic impairment Miglustat has not been evaluated in patients with hepatic impairment. Method of administration Oral use. Orph can be taken with or without food.
4). 4). 1 years. Of these patients, 132 had type 1 Gaucher disease, and 40 had Niemann-Pick type C disease. Adverse reactions were generally of mild to moderate severity and occurred with similar frequency across indications and doses tested.
Tabulated list of adverse reactions Adverse reactions from clinical studies and spontaneous reporting, occurring in > 1% of patients, are listed in the table below by system organ class and frequency (very common: ≥ 1/10, common: ≥ 1/100 to < 1/10, uncommon: ≥ 1/1,000 to < 1/100, rare: ≥ 1/10,000 to < 1/1,000, very rare: < 1/10,000).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness. Table 2. - Tabulated list of adverse reactions System Organ Class (SOC) Frequency Adverse reaction Blood and lymphatic system disorders Common Thrombocytopenia Metabolism and nutrition disorders Very common Weight loss, decreased appetite Psychiatric disorders Common Depression, insomnia, libido decreased Nervous system disorders Very common Tremor Common Peripheral neuropathy, ataxia, amnesia, paraesthesia, hypoaesthesia, headache, dizziness Gastrointestinal disorders Very common Diarrhoea, flatulence, abdominal pain Common Nausea, vomiting, abdominal distension/discomfort, constipation, dyspepsia Musculoskeletal and connective tissue disorder Common Muscle spasms, muscle weakness General disorders and administration site reactions Common Fatigue, asthenia, chills and malaise 7 Investigations Common Nerve conduction studies abnormal Description of selected adverse reactions Weight loss has been reported in 55% of patients.
The greatest prevalence was observed between 6 and 12 months. Miglustat has been studied in indications where certain events reported as adverse reactions, such as neurological and neuropsychological symptoms/signs, cognitive dysfunction and thrombocytopenia could also be due to the underlying conditions.
Tremor 4 Approximately 37% of patients in clinical studies in type 1 Gaucher disease, and 58% of patients in a clinical study in Niemann-Pick type C disease reported tremor on treatment. In type 1 Gaucher disease, these tremors were described as an exaggerated physiological tremor of the hands.
Tremor usually began within the first month of treatment, and in many cases resolved after 1 to 3 months of continued treatment. Dose reduction may ameliorate the tremor, usually within days, but discontinuation of treatment may sometimes be required.
8). The mechanism is most likely inhibition of intestinal disaccharidases such as sucrase-isomaltase in the gastrointestinal tract leading to reduced absorption of dietary disaccharides. In clinical practice, miglustat-induced gastrointestinal events have been observed to respond to individualised diet modification (for example reduction of sucrose, lactose and other carbohydrate intake), to taking miglustat between meals, and/or to anti-diarrhoeal medicinal products such as loperamide.
In some patients, temporary dose reduction may be necessary. Patients with chronic diarrhoea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice.
Miglustat has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease. Orph. Orph. Therefore, in patients with chronic diarrhoea and/or abdominal pain that do not respond to interventions or in the event of clinical worsening, the possibility of Crohn’s disease should be considered.
Effects on spermatogenesis Reliable contraceptive methods should be maintained while male patients are taking miglustat and for 3 months following discontinuation. 3). 3). Special populations Due to limited experience, miglustat should be used with caution in patients with renal or hepatic impairment.
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Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.
2). At present, there is insufficient clinical experience in these patients to provide dosing recommendations. 73 m2) is not recommended. Type 1 Gaucher disease Although no direct comparisons with Enzyme Replacement Therapy (ERT) have been performed in treatment-naive patients with type 1 Gaucher disease, there is no evidence of miglustat having an efficacy or safety advantage over ERT.
1). The efficacy and safety of miglustat has not been specifically evaluated in patients with severe Gaucher disease. Regular monitoring of vitamin B12 level is recommended because of the high prevalence of vitamin B12 deficiency in patients with type 1 Gaucher disease.
5 Cases of peripheral neuropathy have been reported in patients treated with miglustat with or without concurrent conditions such as vitamin B12 deficiency and monoclonal gammopathy. Peripheral neuropathy seems to be more common in patients with type 1 Gaucher disease compared to the general population.
All patients should undergo baseline and repeat neurological evaluation. In patients with type 1 Gaucher disease, monitoring of platelet counts is recommended. Mild reductions in platelet counts without association with bleeding were observed in patients with type 1 Gaucher disease who were switched from ERT to miglustat.
g. every 6 months; continuation of therapy should be re-appraised after at least 1 year of treatment with miglustat. Mild reductions in platelet counts without association to bleeding were observed in some patients with Niemann-Pick type C disease treated with miglustat.
In patients included in the clinical study, 40%- 50% had platelet counts below the lower limit of normal at baseline. Monitoring of platelet counts is recommended in these patients. Reduced growth in the paediatric population Reduced growth has been reported in some paediatric patients with Niemann-Pick type C disease in the early phase of treatment with miglustat where the initial reduced weight gain may be accompanied or followed by reduced height gain.
Growth should be monitored in paediatric and adolescent patients during treatment with miglustat; the benefit/risk balance should be re-assessed on an individual basis for continuation of therapy. Sodium This medicinal product contains less than 1 mmol sodium (23 mg) per hard capsule, that is to say essentially 'sodium-free'.