EU/3/10/753: Orphan designation for the treatment of primary biliary cirrhosis
Table of contents
On 27 July 2010, orphan designation (EU/3/10/753) was granted by the European Commission to Intercept Pharma, Italy, for 6alpha-ethyl-chenodeoxycholic acid (also known as obeticholic acid) for treatment of primary biliary cirrhosis.
6alpha-ethyl-chenodeoxycholic acid has been authorised in the EU as Ocaliva since 12 December 2016.
Treatment of primary biliary cirrhosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Advanz Pharma Limited
Review of designation
On 14 October 2016, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/10/753 for Ocaliva (obeticholic acid1) as an orphan medicinal product for the treatment of primary biliary cirrhosis. 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 primary biliary cirrhosis. The COMP recommended that the orphan designation of the medicine be maintained2.
1Previously known as 6alpha-ethyl-chenodeoxycholic acid.
2The 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.
|November 2022||The sponsorship was transferred to Advanz Pharma Limited, Ireland.|
|April 2019||The sponsorship was transferred to Intercept Pharma International Limited, Ireland.|
|May 2014||The name of the sponsor was corrected to Intercept Italia S.R.L.|
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Ocaliva (obeticholic acid) for the treatment of primary biliary cirrhosis (PDF/80.91 KB)
First published: 19/12/2016
Last updated: 19/12/2016
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
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: