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)

Table of contents

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.

The sponsor’s address was updated in February 2019.

Key facts

Active substance
Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa)
Intented use
Treatment of glycogen storage disease type II (Pompe's disease)
Orphan designation status
Positive
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

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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