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.
pentosan polysulfate sodium
Treatment of mucopolysaccharidosis type I
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|December 2022||The sponsor's address was updated.|
|November 2019||The sponsorship was transferred to Paradigm Biopharmaceuticals (Ireland) Limited.|
Documents related to this orphan designation evaluation
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: