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).

Key facts

Active substance
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

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Amicus Therapeutics UK Ltd
Phoenix House, Oxford Road
Tatling End
Gerrards Cross
Buckinghamshire SL9 7AP
United Kingdom
Tel. + 44 (0)1844 211 669

Patients' organisations

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.

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