Orphacol
What Orphacol is used for
Orphacol is indicated for the treatment of inborn errors in primary bile acid synthesis due to3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency ininfants, children and adolescents aged 1 month to 18 years and adults.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Approved under exceptional circumstances: full data cannot be collected, usually because the disease is very rare.
Key facts
- Brand
- Orphacol
- Active substance
- cholic acid
- Disease area
- Digestive System Diseases; Metabolism, Inborn Errors
- Medicine group
- Bile acids and derivatives
- ATC code
- A05AA03
- Marketing authorisation holder
- Theravia
- Authorised in the EU since
- 2013
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Orphacol used for?
Orphacol is indicated for the treatment of inborn errors in primary bile acid synthesis due to3β-Hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-Oxosteroid-5β-reductase deficiency ininfants, children and adolescents aged 1 month to 18 years and adults.
What is the active substance in Orphacol?
Orphacol contains cholic acid.
Who makes Orphacol?
The EU marketing authorisation holder is Theravia.
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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