EU/3/14/1359: Orphan designation for the treatment of mucopolysaccharidosis type I

pentosan polysulfate sodium


On 19 November 2014, orphan designation (EU/3/14/1359) was granted by the European Commission to Plexcera Therapeutics EU Limited, Ireland, for pentosan polysulfate sodium for the treatment of mucopolysaccharidosis type I.

Key facts

Active substance
pentosan polysulfate sodium
Intended use
Treatment of mucopolysaccharidosis type I
Orphan designation status
EU designation number
Date of designation

Paradigm Biopharmaceuticals (Ireland) Limited 
Fitzwilliam Hall
Fitzwilliam Place
Dublin 2
D02 T292

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

December 2022The sponsor's address was updated.
November 2019The sponsorship was transferred to Paradigm Biopharmaceuticals (Ireland) Limited.

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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