EU/3/01/018: Orphan designation for the treatment of glycogen storage disease type II (Pompe's disease)

Recombinant human acid alpha-glucosidase

Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in March 2016 at the end of the period of market exclusivity.

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.

Key facts

Active substance
Recombinant human acid alpha-glucosidase
Medicine name
Intended use
Treatment of glycogen storage disease type II (Pompe's disease)
Orphan designation status
EU designation number
Date of designation
Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Tel. +31 35 699 1200
Fax +31 35 694 3214

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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