EU/3/17/1863: Orphan designation for the treatment of multiple myeloma

Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains (idecabtagene vicleucel)

Overview

This medicine is now known as idecabtagene vicleucel.

On 20 April 2017, orphan designation (EU/3/17/1863) was granted by the European Commission to bluebird bio France, France, for autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains for the treatment of multiple myeloma.

The sponsorship was transferred to Celgene Europe Limited, United Kingdom, in October 2017 and to Bristol-Myers Squibb Pharma EEIG in November 2021.

The medicinal product has been authorised in the EU as Abecma since 18 August 2021.

Key facts

Active substance
Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains (idecabtagene vicleucel)
Intented use
Treatment of multiple myeloma
Orphan designation status
Positive
EU designation number
EU/3/17/1863
Date of designation
20/04/2017
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 Abecma 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 .

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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