On 12 December 2017, orphan designation (EU/3/17/1949) was granted by the European Commission to IntraBio Ltd, United Kingdom, for acetylleucine (also known as IBI1000) for the treatment of GM2 gangliosidosis.
The sponsorship was transferred to IntraBio Ireland Ltd, Ireland, in April 2019.
|Disease / condition||
Treatment of GM2 gangliosidosis
|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;