EU/3/11/860: Orphan designation for the treatment of Leber's hereditary optic neuropathy

Adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene

Overview

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
Intented use
Treatment of Leber's hereditary optic neuropathy
Orphan designation status
Positive
EU designation number
EU/3/11/860
Date of designation
13/05/2011
Sponsor
Gensight-Biologics
74 rue du Faubourg St Antoine
75012 Paris
France
Tel. +33 1762 17220
E-mail: infos@gensight-biologics.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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.

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:

How useful was this page?

Add your rating