On 17 February 2016, orphan designation (EU/3/16/1612) was granted by the European Commission to Retrophin Europe Limited, Ireland, for methyl 3-((2R)-2-hydroxy-4-(((((S)-1-methoxy-1-oxopropan-2-yl) amino)(phenoxy)phosphoryl)oxy)-3,3-dimethylbutanamido)propanoate for the treatment of pantothenate-kinase-associated neurodegeneration.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in August 2020 on request of the Sponsor.

Key facts

Active substance
Methyl 3-((2R)-2-hydroxy-4-(((((S)-1-methoxy-1-oxopropan-2-yl) amino)(phenoxy)phosphoryl)oxy)-3,3-dimethylbutanamido)propanoate
Disease / condition
Treatment of pantothenate-kinase-associated neurodegeneration
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

Retrophin Europe Limited
2nd Floor, Palmerston House
Fenian Street
Dublin 2
Tel. +353 1 253 0660

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.

How useful was this page?

Add your rating