On 9 January 2020, orphan designation EU/3/19/2241 was granted by the European Commission to ERA Consulting GmbH, Germany, for adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA (also known as ST-920) for the treatment of Fabry disease.
The sponsorship was transferred to YES Pharmaceutical Development Services GmbH, Germany, in August 2021.
adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Treatment of Fabry disease
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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: