EU/3/10/753: Orphan designation for the treatment of primary biliary cirrhosis

6alpha-ethyl-chenodeoxycholic acid


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.

Key facts

Active substance
6alpha-ethyl-chenodeoxycholic acid
Medicine name
Intended use
Treatment of primary biliary cirrhosis
Orphan designation status
EU designation number
Date of designation

Advanz Pharma Limited
Suite 17
Northwood House
Northwood Crescent
Dublin 9
Co. Dublin
D09 V504

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.

Update history

November 2022The sponsorship was transferred to Advanz Pharma Limited, Ireland.
April 2019The sponsorship was transferred to Intercept Pharma International Limited, Ireland.
May 2014The name of the sponsor was corrected to Intercept Italia S.R.L.

Documents related to this orphan designation evaluation

  • List item

    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

  • Patients' organisations

    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:

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