Cerdelga is a medicine used for the long-term treatment of type-1 Gaucher disease in adults. Gaucher disease is a genetic condition, in which a fat called glucosylceramide (or glucocerebroside) builds up in the body, typically in the liver, spleen and bone. This causes symptoms such as anaemia (low red blood cell counts), tiredness, easy bruising, an enlarged spleen and liver, and bone pain and fractures. The disease is caused by the lack of an enzyme for breaking down fat. Gaucher disease is rare, and Cerdelga was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 December 2007.
Therapeutic Indication
### Therapeutic indication Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).
Therapeutic Area (MeSH)
N/AATC Code
A16AX10
ATC Item
eliglustat
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
N/A| Substance | CAS | Monograph |
|---|---|---|
| Eliglustat (tartrate) | N/A | eliglustat (tartrate) |
EMA Name
Cerdelga
Medicine Name
Cerdelga
Aliases
N/ANo risk management plan link.
