EU/3/20/2284: Orphan designation for the treatment of amyotrophic lateral sclerosis

Sodium phenylbutyrate / Tauroursodeoxycholic acid


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.


Key facts

Active substance
  • Sodium phenylbutyrate
  • Tauroursodeoxycholic acid
Intended use
Treatment of amyotrophic lateral sclerosis
Orphan designation status
EU designation number
Date of designation

Barbara Strozzilaan 201
1083 HN Amsterdam

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Update history

The sponsorship was transferred to  Amylyx Pharmaceuticals EMEA B.V., Netherlands , in December 2021.

Documents related to this orphan designation evaluation

Patients' organisations

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:

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