EU/3/16/1650: Orphan designation for the treatment of beta thalassaemia intermedia and major

N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-N-carboxamidomethyl-glycine N-hexadecylamide


Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.

On 28 April 2016, orphan designation (EU/3/16/1650) was granted by the European Commission to QRC Consultants Ltd, United Kingdom, for N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-N-carboxamidomethyl-glycine N-hexadecylamide for the treatment of beta thalassaemia intermedia and major.

Key facts

Active substance
N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-N-carboxamidomethyl-glycine N-hexadecylamide
Intended use
Treatment of beta thalassaemia intermedia and major
Orphan designation status
EU designation number
Date of designation
QRC Consultants Ltd
Silvaco Technology Centre
Compass Point
St Ives
Cambridgeshire PE27 5JL
United Kingdom
Tel. +44 (0)1480 309349
Fax +44 (0)1480 309332

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

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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