EU/3/21/2440 - orphan designation for 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
OrphanHuman

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in October 2024 on request of the Sponsor.

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:

Patients with X-SCID have loss-of-function mutations in the IL2RG gene, which prevent the development of a functional immune system. This medicine is made up of haematopoietic stem cells (HSCs) taken from the patient’s blood. These cells develop into different types of immune cells to help protect from diseases. To make this medicine, the HSCs are genetically modified to contain a working IL2RG gene. When the modified cells are transplanted back into the patient, they are expected to restore normal immune function.

Based on description provided by sponsor

At the time of submission of the application for orphan designation:

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

  • scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
  • market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

  • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

Minutes of the COMP meeting 13-15 April 2021

Adopted Reference Number: EMA/COMP/222143/2021

English (EN) (416.72 KB - PDF)

First published:
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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
Withdrawn
EU designation number
EU/3/21/2440
Date of designation
Sponsor

Transcrip Ireland Limited

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

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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