On 29 April 2009, orphan designation (EU/3/09/623) was granted by the European Commission to EGT San Rocco Italia SRL, Italy, for autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene for the treatment of beta thalassaemia major and intermedia.
Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene
Treatment of beta thalassaemia intermedia and major
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|March 2023||The sponsor’s name was changed from EGT San Rocco Italia SRL to San Rocco Therapeutics Europe S.R.L. and the address was updated|
Documents related to this orphan designation evaluation
EU/3/09/623: Public summary of positive opinion for orphan designation of autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene for the treatment of beta thalassaemi... (PDF/129.73 KB)
First published: 12/05/2009
Last updated: 12/05/2009
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: