On 19 October 2020, orphan designation EU/3/20/2341 was granted by the European Commission to Clinical Technology Centre (Ireland) Limited, Ireland, for autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha (also known as AVR-RD-01) for the treatment of Fabry disease.
Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha
Treatment of Fabry disease
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Review of designation
|June 2022||The sponsorship was transferred to PPD Bulgaria EOOD, Bulgaria in June 2022.|
Documents related to this orphan designation evaluation
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: