On 17 December 2010, orphan designation (EU/3/10/817) was granted by the European Commission to Genable Technologies Ltd, Ireland, for adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked retinitis pigmentosa.
In March 2017, Genable Technologies Ltd changed name to Spark Therapeutics Ireland Ltd.
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2018 on request of the Sponsor.
EU/3/10/817: Public summary of opinion on orphan designation: Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin / adeno-associated viral vector containing a rhodopsin gene for the treatmen... (PDF/151.13 KB)
First published: 19/01/2011
Last updated: 19/01/2011
|Disease / condition||
Treatment of rhodopsin-linked retinitis pigmentosa
|Date of first decision||
|EU designation number||
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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.