EU/3/13/1228: Orphan designation for the treatment of primary sclerosing cholangitis

Obeticholic acid


On 21 March 2014, orphan designation (EU/3/13/1228) was granted by the European Commission to Intercept Italia S.R.L., Italy, for obeticholic acid for the treatment of primary sclerosing cholangitis.

The sponsorship was transferred to Intercept Pharma Ltd, United Kingdom, in June 2016 and to Intercept Pharma International Limited, Ireland, in December 2020.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2022 on request of the Sponsor.

Key facts

Active substance
Obeticholic acid
Intended use
Treatment of primary sclerosing cholangitis
Orphan designation status
EU designation number
Date of designation

Intercept Pharma International Limited
Ormond Building
31-36 Ormond Quay Street
Dublin 7
Tel. +353 1 920 1253

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

Patients' organisations

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:

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