EU/3/16/1714

About

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.

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 decision
22/09/2016
Outcome
Positive
Orphan decision number
EU/3/16/1714

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

IQVIA RDS Ireland Limited
Estuary House
East Point Business Park
Fairview
Dublin 3
Ireland
Tel. +353 1819 5100

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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