Products
Miglustat is commercially available in capsule form (Zavesca, generic). It has been approved in many countries since 2004.
Structure and properties
Miglustat (C10H21NO4, Mr = 219.3 g/mol) is an -alkylated iminosugar. It exists as a white crystalline powder with a bitter taste that is readily soluble in water.
Effects
Miglustat (ATC A16AX06) is an inhibitor of the enzyme glucosylceramide synthase. This reduces the formation and accumulation of glucocerebroside (glucosylceramide). Gaucher disease is characterized by a deficiency of the lysosomal enzyme beta-glucocerebrosidase, which degrades glucocerebroside into glucose and ceramide. This leads to accumulation of glucocerebroside in cells, mainly in macrophages.
Indications
- Gaucher disease type 1
- Niemann-Pick disease type C (progressive neurological manifestations).
Dosage
According to the professional information. The capsules are usually taken three times a day and independently of meals.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Adverse effects
The most common possible adverse effects include diarrhea, flatulence, abdominal pain, weight loss, loss of appetite, visual disturbances, and tremor.
