Cerdelga is a medicine used for the long\-term treatment of type 1 Gaucher disease in adults and in children from 6 years of age, weighing at least 15 kg, whose disease is well controlled by enzyme replacement therapy (ERT). 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 levels of red blood cells), tiredness, easy bruising, an enlarged spleen and liver, and bone pain and fractures. The disease is caused by the lack of an enzyme responsible 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\. Further information on the orphan designation can be found on the EMA [website](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-514). Cerdelga contains the active substance eliglustat.
Therapeutic Indication
**Adults** 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). **Paediatric population (from 6 to \< 18 years of age) weighing ≥ 15 kg** Cerdelga is indicated for paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who are stable on enzyme replacement therapy (ERT), and who are CYP2D6 PMs, IMs or EMs.
Therapeutic Area (MeSH)
ATC Code
A16AX10
ATC Item
N/A
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
EMA Name
Cerdelga
Medicine Name
Cerdelga
Aliases
N/ANo risk management plan link.
