On 20 March 2017, orphan designation (EU/3/17/1849) was granted by the European Commission to Freeline Therapeutics Ltd, United Kingdom, for adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene for treatment of Fabry disease.
The sponsor's address was updated in July 2018.
The sponsorship was transferred to Freeline Therapeutics (Ireland) Limited Ireland in May 2019.
Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene
|Disease / condition||
Treatment of Fabry disease
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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;