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.
Alpha galactosidase A
Treatment of Fabry disease
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Review of designation
Documents related to this orphan designation evaluation
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: