EU/3/20/2321: Orphan designation for the treatment of Wilson's disease

Adeno-associated viral vector serotype 3B encoding shortened human ATP7B

Overview

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
Intended use
Treatment of Wilson's disease
Orphan designation status
Positive
EU designation number
EU/3/20/2321
Date of designation
21/08/2020
Sponsor

Vivet Therapeutics SAS
80 boulevard Haussmann 
75008 Paris
France
Tel. +33 6 98 79 97 31
E-mail: info@vivet-therapeutics.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:

  • 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:

How useful was this page?

Add your rating