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).

The sponsorship was transferred to Amicus Therapeutics Europe Limited, Ireland, in March 2019.

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 Europe Limited
Block 1, Blanchardstown Corporate Park
Ballycoolen Road
Dublin D15 AKK1
Tel. +353 1 588 6850

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;

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