On 13 May 2011, orphan designation (EU/3/11/860) was granted by the European Commission to Institut de la Vision, France, for adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene for treatment of Leber's hereditary optic neuropathy.
The sponsorship was transferred to Gensight-Biologics, France, in September 2013.
EU/3/11/860: Public summary of opinion on orphan designation: Adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene for the treatment of Leber's hereditary optic neuropathy (PDF/132.6 KB)
First published: 27/05/2011
Last updated: 07/10/2013
EMA/COMP/154900/2011 Rev. 1
Adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene
|Disease / condition||
Treatment of Leber's hereditary optic neuropathy
|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:
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.