EU/3/12/957

About

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
Disease / condition
Treatment of X-linked chronic granulomatous disease
Date of first decision
09/02/2012
Outcome
Positive
EU designation number
EU/3/12/957

Review of designation

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

Sponsor's contact details

Orchard Therapeutics (Netherlands) B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
The Netherlands
Tel. +31 6570 41430
E-mail: info@orchard-tx.com

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;

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