cholic acid

This medicine is authorised for use in the European Union.


This is a summary of the European public assessment report (EPAR) for Kolbam. 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 Kolbam.

For practical information about using Kolbam, patients should read the package leaflet or contact their doctor or pharmacist.

This EPAR was last updated on 20/12/2019

Authorisation details

Product details
Agency product number
Active substance
cholic acid
International non-proprietary name (INN) or common name
cholic acid
Therapeutic area (MeSH)
Metabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Exceptional circumstancesExceptional circumstances

This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Retrophin Europe Ltd
Date of issue of marketing authorisation valid throughout the European Union
Contact address
Palmerston House
Fenian Street
Dublin 2

Product information

14/11/2019 Kolbam - EMEA/H/C/002081 - EMEA/H/C/002081/II/0028


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Pharmacotherapeutic group

Bile and liver therapy

Therapeutic indication

Cholic Acid FGK is indicated for the treatment of inborn errors of primary bile acid synthesis, in infants from one month of age for continuous lifelong treatment through adulthood, encompassing the following single enzyme defects:

  • sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency;
  • 2- (or alpha-) methylacyl-CoA racemase (AMACR) deficiency;
  • cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.

Assessment history

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