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.
Adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene
Treatment of Leber's hereditary optic neuropathy
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Review of designation
Documents related to this orphan designation evaluation
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
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.
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: