EU/3/16/1726: Orphan designation for the treatment of glycogen storage disease type II (Pompe's disease)

Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues

Overview

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
Intended use
Treatment of glycogen storage disease type II (Pompe's disease)
Orphan designation status
Positive
EU designation number
EU/3/16/1726
Date of designation
29/08/2016
Sponsor
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

Review of designation

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

Documents related to this orphan designation evaluation

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.

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:

How useful was this page?

Add your rating