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.

Key facts

Active substance
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

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

Sponsor's contact details

Comet Therapeutics
Galileiweg 8
2333 BD Leiden
The Netherlands

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.

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