On 22 May 2017, orphan designation (EU/3/17/1878) was granted by the European Commission to IntraBio Ltd, United Kingdom, for ursodeoxycholic acid for the treatment of Niemann-Pick disease.
The sponsorship was transferred to IntraBio Ireland Ltd, Ireland, in April 2019.
Treatment of Niemann-Pick disease
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
Documents related to this orphan designation evaluation
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: