EU/3/20/2396: Orphan designation for the treatment of Fabry disease

Alpha galactosidase A

Overview

On 6 January 2021, orphan designation EU/3/20/2396 was granted by the European Commission to Consejo Superior de Investigaciones Científicas, Spain, for alpha galactosidase A (also known as rh GLA) for the treatment of Fabry disease.

Key facts

Active substance
Alpha galactosidase A
Intended use
Treatment of Fabry disease
Orphan designation status
Positive
EU designation number
EU/3/20/2396
Date of designation
06/01/2021
Sponsor

Consejo Superior De Investigaciones Cientificas
Calle Serrano 142
28006 Madrid
Spain
Tel: +915681488
E-mail: patentes@csic.es

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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