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.
Kolbam : EPAR - Risk-management-plan summary (PDF/57.93 KB)
First published: 09/12/2015
Last updated: 04/02/2016
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Metabolism, Inborn Errors
|Anatomical therapeutic chemical (ATC) code||
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.
Retrophin Europe Ltd
|Date of issue of marketing authorisation valid throughout the European Union||
07/12/2016 Kolbam - EMEA/H/C/002081 - IB/0019
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
Please note that the size of the above document can exceed 50 pages.
You are therefore advised to be selective about which sections or pages you wish to print.
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.
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 September 201525/09/2015
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 January 201424/01/2014
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 18-21 November 201322/11/2013