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.

Key facts

Active substance
6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate
Disease / condition
Treatment of mucopolysaccharidosis type I
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

TMC Pharma (EU) Limited
The Black Church
St. Mary's Place
Dublin D07 P4AX
Tel. +353 76 670 5745

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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