On 28 October 2009, orphan designation (EU/3/09/683) was granted by the European Commission to Special Products Ltd, United Kingdom, for cholic acid for the treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid.
The sponsorship was transferred to Retrophin Europe Limited, Ireland, in May 2015.
Cholic acid was authorised in the EU as Kolbam on 20 November 2015.
The marketing authorisation was withdrawn on 15 July 2020.
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in July 2020 at the time of withdrawal of the marketing authorisation.
Treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid
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Review of designation
During its meeting of 6 to 8 October 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/09/683 for Kolbam (cholic acid) as an orphan medicinal product for the treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained1.
1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.
Documents related to this orphan designation evaluation
EU/3/09/683: Public summary of positive opinion for orphan designation of cholic acid for the treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid (PDF/217.46 KB)
First published: 18/11/2009
Last updated: 28/09/2015
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Kolbam (cholic acid) for the treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid (PDF/146.72 KB)
First published: 07/01/2016
Last updated: 07/01/2016
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: