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.
Orphacol : EPAR - Summary for the public (PDF/85.95 KB)
First published: 02/10/2013
Last updated: 02/10/2013
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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.
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04/08/2017 Orphacol - EMEA/H/C/001250 - IA/0021
- 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.
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 one month to 18 years and adults.