EU/3/14/1406: Orphan designation for the treatment of inborn errors in primary bile acid synthesis
Table of contents
On 16 December 2014, orphan designation (EU/3/14/1406) was granted by the European Commission to Sigma-Tau Pharma Ltd, United Kingdom, for chenodeoxycholic acid for the treatment of inborn errors in primary bile acid synthesis.
The sponsorship was transferred to sigma-tau Arzneimittel GmbH, Germany, in May 2015.
Chenodeoxycholic acid has been authorised in the EU as Chenodeoxycholic acid sigma-tau since 10 April 2017.
The name of the product changed to Chenodeoxycholic acid Leadiant in May 2017.
The sponsorship was transferred to Leadiant GmbH, Germany, in June 2017.
Chenodeoxycholic acid sigma-tau
Treatment of inborn errors in primary bile acid synthesis
|Orphan designation status||
|EU designation number||
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Review of designation
On 16 February 2017, the Committee for Orphan Medicinal Products (COMP) concluded its review of the designation EU/3/14/1406 for Chenodeoxycholic acid sigma-tau (chenodeoxycholic acid) as an orphan medicinal product for the treatment of inborn errors in primary bile acid synthesis. 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 are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with inborn errors in primary bile acid synthesis. The COMP recommended that the orphan designation of the medicine be maintained1.
1 The 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 the same therapeutic indication cannot be placed on the market.
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Chenodeoxycholic acid sigma-tau (chenodeoxycholic acid) for the treatment of inborn errors in primary bile acid synthesis (PDF/90.96 KB)
First published: 11/05/2017
Last updated: 29/06/2017
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.
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: