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.
The sponsorship was transferred to Quintiles Ireland Limited, Ireland, in March 2018.
In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.
The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in August 2020.
|Disease / condition||
Treatment of mucopolysaccharidosis type I
|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.