On 27 October 2011, orphan designation (EU/3/11/921) was granted by the European Commission to BioMarin Europe Ltd, United Kingdom, for GILT-tagged recombinant human acid alpha glucosidase for the treatment of glycogen storage disease type II (Pompe's disease).
The sponsorship was transferred to Biomarin International Limited., Ireland, in November 2021.
Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase
Treatment of glycogen storage disease type II (Pompe's disease)
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Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Documents related to this orphan designation evaluation
EU/03/11/921: Public summary of opinion on orphan designation: Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase for the treatment of glycogen storage disease type II (P... (PDF/138.62 KB)
First published: 23/11/2011
Last updated: 23/11/2011
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: