On 21 March 2018, orphan designation (EU/3/18/2000) was granted by the European Commission to Amicus Therapeutics UK Ltd, United Kingdom, for recombinant human acid alpha-glucosidase (also known as ATB200) for the treatment of glycogen storage disease type II (Pompe's disease).
Recombinant human acid alpha-glucosidase
|Disease / condition||
Treatment of glycogen storage disease type II (Pompe's disease)
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.