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
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Review of designation
Sponsor's contact details
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.