EU/3/14/1300: Orphan designation for the treatment of beta thalassaemia intermedia and major

Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept)

Overview

On 29 July 2014, orphan designation (EU/3/14/1300) was granted by the European Commission to IDEA Innovative Drug European Associates Limited, the United Kingdom, for recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain for the treatment of beta thalassaemia intermedia and major.

This medicine is now known as luspatercept.

The sponsorship was transferred to Celgene Europe Limited, United Kingdom, in February 2015. The sponsorship was transferred to Celgene Europe B.V., The Netherlands, in September 2018.

Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain for treatment of beta thalassaemia intermedia and major has been authorised in the EU as Reblozyl since 25 June 2020. 

The sponsorship was transferred to Bristol-Myers Squibb Pharma EEIG, Ireland in February 2021.

Key facts

Active substance
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept)
Intended use
Treatment of beta thalassaemia intermedia and major
Orphan designation status
Positive
EU designation number
EU/3/14/1300
Date of designation
29/07/2014
Sponsor

Bristol-Myers Squibb Pharma EEIG
Plaza 254
Blanchardstown Corporate Park 2
Dublin 15
D15 T867
Ireland
Tel. +353 1 483 3857
E-mail: medical.information@bms.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Reblozyl at the time of change to the terms of the marketing authorisation, and confirmed that the orphan designation should be maintained: PDF icon Orphan maintenance assessment report

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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