EU/3/14/1251

Table of contents

About

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)
Disease / condition
Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision
26/03/2014
Outcome
Positive
EU designation number
EU/3/14/1251

Sponsor's contact details

Genzyme Europe B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Nederlands
Tel. +31 20 245 4000
E-mail: eumedinfo.gz@sanofi.com

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;

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