This is a summary of the European public assessment report (EPAR) for Yargesa. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Yargesa. For practical information about using Yargesa, patients should read the package leaflet or contact their doctor or pharmacist.
Therapeutic Indication
### Therapeutic indication Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
Therapeutic Area (MeSH)
N/AATC Code
A16AX06
ATC Item
miglustat
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
N/A| Substance | CAS | Monograph |
|---|---|---|
| miglustat | N/A | 麦格司他 |
EMA Name
Yargesa
Medicine Name
Yargesa
Aliases
N/ANo risk management plan link.
