EU/3/18/2058

About

On 24 August 2018, orphan designation (EU/3/18/2058) was granted by the European Commission to IQVIA RDS Ireland Limited, Ireland, for 1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt (also known as hepcidin mimetic) for the treatment of beta-thalassaemia intermedia and major.

Key facts

Active substance
1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt
Disease / condition
Treatment of beta thalassaemia intermedia and major
Date of first decision
24/08/2018
Outcome
Positive
EU designation number
EU/3/18/2058

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 1 819 51 00
E-mail: Malia.Segar@iqvia.com

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;

How useful was this page?

Add your rating