On 28 April 2016, orphan designation (EU/3/16/1654) was granted by the European Commission to Acies Bio d.o.o., Slovenia, for S-acetyl-(S)-4'-phosphopantetheine, calcium salt for the treatment of pantothenate-kinase-associated neurodegeneration.
The sponsorship was transferred to TM3 Therapeutics B.V., The Netherlands, in September 2018.
The sponsor’s name was updated in July 2019.
S-acetyl-(S)-4'-phosphopantetheine, calcium salt
|Disease / condition||
Treatment of pantothenate-kinase-associated neurodegeneration
|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.