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.
Update: Cholic acid (Kolbam) has been authorised in the EU since 20 November 2015 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.
More information on Kolbam can be found in the European public assessment report (EPAR) on the Agency's website.
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/126.45 KB)
First published: 18/11/2009
Last updated: 28/09/2015
|Disease / condition||
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.
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/92.78 KB)
First published: 07/01/2016
Last updated: 07/01/2016
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.