EU/3/04/233: Orphan designation for the treatment of gamma sarcoglycanopathy

Adeno-associated viral vector containing the human gamma-sarcoglycan gene


Please note that this product was withdrawn from the Community register of designated orphan medicinal products in May 2014 on request of the sponsor.

On 21 October 2004, orphan designation (EU/3/04/233) was granted by the European Commission to Généthon, France, for adeno-associated viral vector containing the human gamma sarcoglycan gene for the treatment of gamma sarcoglycanopathy.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Key facts

Active substance
Adeno-associated viral vector containing the human gamma-sarcoglycan gene
Intended use
Treatment of gamma sarcoglycanopathy
Orphan designation status
EU designation number
Date of designation
1 bis rue de l’internationale
F-91000 Evry
Telephone: +33 1 69 47 29 17
Telefax: +33 1 69 47 19 46

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:

  • 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.

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:

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