EU/3/18/2072

About

On 26 October 2018, orphan designation (EU/3/18/2072) was granted by the European Commission to FGK Representative Service GmbH, Germany, for 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate (also known as ELX-02) for the treatment of cystic fibrosis.

The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in October 2020.

Key facts

Active substance
6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate
Disease / condition
Treatment of cystic fibrosis
Date of first decision
26/10/2018
Outcome
Positive
EU designation number
EU/3/18/2072

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
Ireland
Tel. +353 76 670 5745
E-mail: regulatory.services@tmcpharma.com

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.

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