On 21 August 2020, orphan designation EU/3/20/2321 was granted by the European Commission to Vivet Therapeutics S.A.S., France, for adeno-associated viral vector serotype 3B encoding shortened human ATP7B (also known as VTX-801) for the treatment of Wilson’s disease.

Key facts

Active substance
Adeno-associated viral vector serotype 3B encoding shortened human ATP7B
Disease / condition
Treatment of Wilson's disease
Date of first decision
EU designation number

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

Vivet Therapeutics SAS
80 boulevard Haussmann 
75008 Paris
Tel. +33 6 98 79 97 31

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.

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