EU/3/14/1331

About

On 22 August 2014, orphan designation (EU/3/14/1331) 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 myelodysplastic syndromes.

This medicine is now known as luspatercept.

The sponsorship was transferred to Celgene Europe Limited, United Kingdom, in June 2015 and subsequently 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 myelodysplastic syndromes 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)
Disease / condition
Treatment of myelodysplastic syndromes
Date of first decision
22/08/2014
Outcome
Positive
EU designation number
EU/3/14/1331

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Reblozyl at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. 

More information is available in the PDF icon orphan medicine assessment report .

Sponsor's contact details

Bristol-Myers Squibb Pharma EEIG
Plaza 254
Blanchardstown Corporate Park 2
Dublin 15
D15 T867
Ireland
Tel. +44 (0) 208 831 8327
E-mail: medical.information@bms.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.

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