Gaucher disease type 1

Active Ingredient: Miglustat

Indication for Miglustat

Population group: only adults (18 - 65 years old)
Therapeutic intent: Curative procedure

Miglustat is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.

For this indication, competent medicine agencies globally authorize below treatments:

100 mg 3 times a day


Dosage regimens

Oral, 100 milligrams miglustat, 3 times daily.

Detailed description

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.

Dosage considerations

Miglustat can be taken with or without food.

Active ingredient


Miglustat is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycolipids, and a pharmacokinetic enzyme stabiliser of cipaglucosidase alfa. Miglustat is indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease and for the treatment of progressive neurological manifestations in patients with Niemann-Pick type C disease. Also, miglustat must be used in combination with cipaglucosidase alfa for long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

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