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
|Disease / condition||
Treatment of glycogen storage disease type II (Pompe's disease)
|Date of decision||
|Orphan decision number||
Sponsor's contact details
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.