EU/3/18/2129: Orphan designation for the treatment of glycogen storage disease type II (Pompe's disease)
Table of contents
On 11 January 2019, orphan designation (EU/3/18/2129) was granted by the European Commission to Amicus Therapeutics UK Limited, United Kingdom, for miglustat for the treatment of glycogen storage disease type II (Pompe's disease).
Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2023 on request of the Sponsor.
Treatment of glycogen storage disease type II (Pompe's disease)
|Orphan designation status||
|EU designation number||
|Date of designation||
Amicus Therapeutics Europe Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
|March 2023||The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.|
|March 2019||The sponsorship was transferred to Amicus Therapeutics Europe Limited, Ireland.|
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: