EU/3/21/2464: Orphan designation for the treatment of follicular lymphoma
Table of contents
This medicine was designated as an orphan medicine for the treatment of follicular lymphoma in the European Union on 19 July 2021.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Treatment of follicular lymphoma
|Orphan designation status||
|EU designation number||
|Date of designation||
Novartis Europharm Limited
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Kymriah at the time of change to the terms of the marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the orphan medicine assessment report .
|April 2022||Tisagenlecleucel for treatment of follicular lymphoma has been authorised in the EU as Kymriah since 29 April 2022.|
Documents related to this orphan designation evaluation
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: