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.
Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene
Treatment of X-linked chronic granulomatous disease
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Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
|July 2022||The sponsor's address was updated in July 2022.|
Documents related to this orphan designation evaluation
EU/3/12/957: Public summary of opinion on orphan designation: Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene for the treatment of X-linked chronic granu... (PDF/137.16 KB)
First published: 01/03/2012
Last updated: 01/03/2012
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: