On 14 February 2001, orphan designation (EU/3/01/018) was granted by the European Commission to Genzyme B.V., The Netherlands, for recombinant human acid α-glucosidase for the treatment of glycogen storage disease type II (Pompe's disease).
The sponsorship was transferred to Genzyme Europe BV, The Netherlands, in April 2002.
Update: recombinant human acid α-glucosidase has been authorised in the EU as Myozyme since 29 March 2006.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Recombinant human acid alpha-glucosidase
Treatment of glycogen storage disease type II (Pompe's disease)
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Documents related to this orphan designation evaluation
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: