EU/3/12/957: Orphan designation for the treatment of X-linked chronic granulomatous disease

Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene

Overview

On 9 February 2012, orphan designation (EU/3/12/957) was granted by the European Commission to Généthon, France, for autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene for the treatment of X-linked chronic granulomatous disease.

The sponsorship was transferred to Orchard Therapeutics (Netherlands) B.V, Netherlands, in September 2019.

Key facts

Active substance
Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene
Intended use
Treatment of X-linked chronic granulomatous disease
Orphan designation status
Positive
EU designation number
EU/3/12/957
Date of designation
09/02/2012
Sponsor

Orchard Therapeutics (Netherlands) B.V.
Basisweg 10
Noord-Holland
1043 AP Amsterdam
Netherlands
E-mail:  regulatory@orchard-tx.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Update history

DATE Update
July 2022 The sponsor's address was updated in July 2022.

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:

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