On 22 September 2016, orphan designation (EU/3/16/1714) was granted by the European Commission to Coté Orphan Consulting UK Limited, United Kingdom, for 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate (also known as ELX-02) for the treatment of mucopolysaccharidosis type I.
Treatment of mucopolysaccharidosis type I
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|May 2022||The sponsor’s address was updated in May 2022.|
|March 2021||The sponsor’s address was updated in March 2021.|
|August 2020||The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in August 2020.|
|May 2018||In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.|
|March 2018||The sponsorship was transferred to Quintiles Ireland Limited, Ireland, in March 2018.|
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: