EU/3/21/2440: Orphan designation for the treatment of X-linked severe combined immunodeficiency
Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene
Overview
This medicine was designated as an orphan medicine for the treatment of X-linked severe combined immunodeficiency in the European Union on 20 May 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:
Key facts
Active substance |
Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene
|
Intended use |
Treatment of X-linked severe combined immunodeficiency
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/21/2440
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Date of designation |
20/05/2021
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Sponsor |
Real Regulatory Limited |
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: