This is a summary of the European public assessment report (EPAR) for Orphacol. It explains how the European Medicines Agency assessed the medicine to recommend its authorisation in the European Union (EU) and its conditions of use. It is not intended to provide practical advice on how to use Orphacol. For practical information about using Orphacol, patients should read the package leaflet or contact their doctor or pharmacist.
Therapeutic Indication
### Therapeutic indication Orphacol is indicated for the treatment of inborn errors in primary bile acid synthesis due to 3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency in infants, children and adolescents aged 1 month to 18 years and adults.
Therapeutic Area (MeSH)
ATC Code
A05AA03
ATC Item
cholic acid
Pharmacotherapeutic Group
Bile acids and derivatives
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| cholic acid | N/A | 胆酸；胆汁酸 |
EMA Name
Orphacol
Medicine Name
Orphacol
Aliases
N/A