EU/3/14/1251: Orphan designation for the treatment of glycogen storage disease type II (Pompe's disease)

Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa)

Overview

On 26 March 2014, orphan designation (EU/3/14/1251) was granted by the European Commission to Genzyme Europe BV, the Netherlands, for recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan for the treatment of glycogen storage disease type II (Pompe's disease).

This medicine is now known as avalglucosidase alfa.

Please note that this product (marketed as Nexviadyme) was withdrawn from the Community register of orphan medicinal products by the European Commission in April 2022 at the time of the granting of a marketing authorisation.

 

Key facts

Active substance
Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa)
Intended use
Treatment of glycogen storage disease type II (Pompe's disease)
Orphan designation status
Withdrawn
EU designation number
EU/3/14/1251
Date of designation
26/03/2014
Sponsor

Genzyme Europe B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Nederlands
Tel. +31 2024 54000
E-mail: eumedinfo.gz@sanofi.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Nexviadyme at the time of marketing authorisation, and recommended that the orphan designation should not be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Update history

DateUpdate
April 2022Product withdrawn from the Community register of orphan medicinal products
February 2019The sponsor’s address was updated in February 2019.

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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