EU/3/16/1726

About

On 29 August 2016, orphan designation (EU/3/16/1726) was granted by the European Commission to NanoMedSyn, France, for recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues for the treatment of glycogen storage disease type II (Pompe's disease).

Key facts

Active substance
Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues
Disease / condition
Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision
29/08/2016
Outcome
Positive
EU designation number
EU/3/16/1726

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

NanoMedSyn
15 Avenue Charles Flahault
Faculté de Pharmacie
Bât E 2ème étage
34093 Montpellier cedex 5
France
Tel. +33 4 11 75 96 19
E-mail: contact@nanomedsyn.com

Patients' organisations

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.

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