Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in June 2014 on request of the Sponsor.
On 6 February 2009, orphan designation (EU/3/08/609) was granted by the European Commission to Fondazione Telethon, Italy, for adeno-associated viral vector serotype 5 containing the human ABCA4 gene for the treatment of Stargardt's disease.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
EU/3/08/609: Public summary of positive opinion for orphan designation of adeno-associated viral vector serotype 5 containing the human ABCA4 gene for the treatment of Stargardt’s disease (PDF/126.34 KB)
First published: 20/03/2009
Last updated: 16/10/2014
Adeno-associated viral vector serotype 5 containing the human ABCA4 gene
|Disease / condition||
Treatment of Stargardt's disease
|Date of first decision||
|EU designation number||
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.