On 20 March 2017, orphan designation (EU/3/17/1848) was granted by the European Commission to IntraBio Ltd, United Kingdom, for acetylleucine for the treatment of Niemann-Pick disease.
The sponsorship was transferred to IntraBio Ireland Ltd, Ireland, in April 2019.
|Disease / condition||
Treatment of Niemann-Pick disease
|Date of first decision||
|EU designation number||
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;