EU/3/20/2396: Orphan designation for the treatment of Fabry disease
Alpha galactosidase A
Table of contents
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 |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: