EU/3/20/2252: Orphan designation for the treatment of multiple myeloma

Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen

Overview

On 28 February 2020, orphan designation EU/3/20/2252 was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen (also known as JNJ-68284528 or LCAR-B38M CAR-T cells) for the treatment of multiple myeloma.

This medicine is now known as ciltacabtagene autoleucel.

The medicinal product has been authorised in the EU as Carvykti since 25 May 2022.

Key facts

Active substance
Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen
Intended use
Treatment of multiple myeloma
Orphan designation status
Positive
EU designation number
EU/3/20/2252
Date of designation
28/02/2020
Sponsor

Janssen-Cilag International N.V.
Turnhoutseweg 30
2340 Beerse
Antwerp
Belgium
Tel: +31621385718
E-mail: nreymond@its.jnj.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Carvykti 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

Update history

DateUpdate
May 2022The medicinal product has been authorised in the EU as Carvykti since 25 May 2022.  

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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