Products
Eliglustat is commercially available in the form of hard capsules (Cerdelga). It was approved in many countries in 2020.
Structure and properties
Eliglustat (C23H36N2O4, Mr = 404.5 g/mol) is present in the drug as eliglustat tartrate. Eliglustat is an analog of glucocerebroside.
Effects
Eliglustat (ATC A16AX10) is a specific 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
For long-term treatment of adult patients with Gaucher disease type 1.
Dosage
According to the SmPC. Depending on CYP2D6 activity, capsules are taken once or twice daily, independent of meals.
Contraindications
- Hypersensitivity
- Treatment with CYP inhibitors (depending on CYP2D6 activity, see FI).
For full precautions, see the drug label.
Interactions
Eliglustat is a substrate of CYP2D6 and CYP3A4 and corresponding drug-drug interactions are possible.
Adverse effects
The most common possible adverse effect of treatment is diarrhea.
