Active Ingredient: Miglustat
Miglustat is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Oral, 200 milligrams miglustat, 3 times daily.
The recommended dose for the treatment of adult and adolescent patients (12 years of age and above) with Niemann-Pick type C disease is 200 mg three times a day.
Miglustat can be taken with or without food.
For:
Regimen A: In case that patient body surface area is ≥ 1.25 m², oral, 200 milligrams miglustat, 3 times daily.
Regimen B: In case that patient body surface area is ≥ 0.88 m² and patient body surface area is ≤ 1.25 m², oral, 200 milligrams miglustat, 2 times daily.
Regimen C: In case that patient body surface area is ≥ 0.73 m² and patient body surface area is ≤ 0.88 m², oral, 100 milligrams miglustat, 3 times daily.
Regimen D: In case that patient body surface area is ≥ 0.47 m² and patient body surface area is ≤ 0.73 m², oral, 100 milligrams miglustat, 2 times daily.
Regimen E: In case that patient body surface area is ≤ 0.47 m², oral, 100 milligrams miglustat, once daily.
Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below:
| Body surface area (m²) | Recommended dose |
|---|---|
| >1.25 | 200 mg three times a day |
| >0.88-1.25 | 200 mg twice a day |
| >0.73-0.88 | 100 mg three times a day |
| >0.47-0.73 | 100 mg twice a day |
| ≤0.47 | 100 mg once a day |
Temporary dose reduction may be necessary in some patients because of diarrhoea.
The benefit to the patient of treatment with miglustat should be evaluated on a regular basis.
There is limited experience with the use of miglustat in Niemann-Pick type C disease patients under the age of 4 years.
Miglustat can be taken with or without food.
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