On 13 November 2019, orphan designation EU/3/19/2218 was granted by the European Commission to Idorsia Pharmaceuticals Deutschland GmbH, Germany, for (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol (also known as ACT-519276) for the treatment of GM2 gangliosidosis.
|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.