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
Table of contents
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.
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: