EU/3/11/921: Orphan designation for the treatment of glycogen storage disease type II (Pompe's disease)

Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase

Overview

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.

Key facts

Active substance
Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase
Intented use
Treatment of glycogen storage disease type II (Pompe's disease)
Orphan designation status
Positive
EU designation number
EU/3/11/921
Date of designation
27/10/2011
Sponsor

Biomarin International Limited
Shanbally
Ringaskiddy 
Co. Cork
P43 R298
Ireland
Email: shanbally@bmrn.com
 

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

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.

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:

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