EU/3/16/1745: Orphan designation for the treatment of diffuse large B-cell lymphoma

Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel)

Overview

On 14 October 2016, orphan designation (EU/3/16/1745) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (also known as CTL019) for the treatment of diffuse large B-cell lymphoma.

This medicine is now known as tisagenlecleucel.

The sponsorship was transferred to Novartis Europharm Limited, Ireland, in May 2018.

Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 for treatment of diffuse large B-cell lymphoma has been authorised in the EU as Kymriah since 23 August 2018.

Key facts

Active substance
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel)
Medicine name
Kymriah
Intended use
Treatment of diffuse large B-cell lymphoma
Orphan designation status
Positive
EU designation number
EU/3/16/1745
Date of designation
14/10/2016
Sponsor

Novartis Europharm Limited
Vista Building
Elm Park       
Merrion Road
Dublin 4 D04 A9N6
Tel. +41 613 241 111
E-mail: orphan.enquiries@novartis.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Kymriah 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:

How useful was this page?

Add your rating
Average
1 rating
1 rating