EU/3/16/1650: Orphan designation for the treatment of beta thalassaemia intermedia and major
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.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.
Treatment of beta thalassaemia intermedia and major
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Review of designation
Documents related to this orphan designation evaluation
EU/3/16/1650: Public summary of opinion on orphan designation: 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... (PDF/157.09 KB)
First published: 30/05/2016
Last updated: 10/08/2017
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: