On 4 June 2020, orphan designation EU/3/20/2284 was granted by the European Commission to Drug Development and Regulation S.L., Spain, for sodium phenylbutyrate, tauroursodeoxycholic acid (also known as AMX0035) for the treatment of amyotrophic lateral sclerosis.
Treatment of amyotrophic lateral sclerosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
The sponsorship was transferred to Amylyx Pharmaceuticals EMEA B.V., Netherlands , in December 2021.
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: