EU/3/19/2241: Orphan designation for the treatment of Fabry disease

adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA

Table of contents

Overview

On 9 January 2020, orphan designation EU/3/19/2241 was granted by the European Commission to ERA Consulting GmbH, Germany, for adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA (also known as ST-920) for the treatment of Fabry disease.

The sponsorship was transferred to YES Pharmaceutical Development Services GmbH, Germany, in August 2021.

Key facts

Active substance
adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Intended use
Treatment of Fabry disease
Orphan designation status
Positive
EU designation number
EU/3/19/2241
Date of designation
09/01/2020
Sponsor

YES Pharmaceutical Development Services GmbH
Bahnstrasse 42-46
61381 Friedrichsdorf
Hassia
Germany
Tel. +49 6172 764 64 65
E-mail: communications@sangamo.com

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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