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
Table of contents
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. |
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
orphan medicine assessment report
.
Update history
Date | Update |
---|---|
May 2022 | The medicinal product has been authorised in the EU as Carvykti since 25 May 2022. |
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: