EU/3/16/1714: Orphan designation for the treatment of mucopolysaccharidosis type I

6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate


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.

Key facts

Active substance
6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate
Intended use
Treatment of mucopolysaccharidosis type I
Orphan designation status
EU designation number
Date of designation

TMC Pharma (EU) Limited
7A Durands Court
45 Parnell Street
Co. Waterford
X91 P381
Tel. + 353766705745

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

May 2022The sponsor’s address was updated in May 2022.
March 2021The sponsor’s address was updated in March 2021.
August 2020The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in August 2020.
May 2018In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.
March 2018The sponsorship was transferred to Quintiles Ireland Limited, Ireland, in March 2018.

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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