EU/3/11/860

About

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.

Key facts

Active substance
Adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene
Disease / condition
Treatment of Leber's hereditary optic neuropathy
Date of first decision
13/05/2011
Outcome
Positive
EU designation number
EU/3/11/860

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

Gensight-Biologics
74 rue du Faubourg St Antoine
75012 Paris
France
Tel. +33 1762 17220
E-mail: infos@gensight-biologics.com

Patients' organisations

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.

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