EU/3/12/1094: Orphan designation for the treatment of achondroplasia
Modified recombinant human C-type natriuretic peptide (Vosoritide)
Table of contents
Overview
On 24 January 2013, orphan designation (EU/3/12/1094) was granted by the European Commission to BioMarin Europe Ltd, United Kingdom, for modified recombinant human C-type natriuretic peptide for the treatment of achondroplasia.
The sponsorship was transferred to BioMarin International Limited, Ireland, in February 2019.
This medicine is now known as Vosoritide.
The medicinal product has been authorised in the EU as Voxzogo since 26 August 2021.
Key facts
Active substance |
Modified recombinant human C-type natriuretic peptide (Vosoritide)
|
Intended use |
Treatment of achondroplasia
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/12/1094
|
Date of designation |
24/01/2013
|
Sponsor |
BioMarin International Limited |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
The Committee for Orphan Medicinal Products reviewed the orphan designation of product at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Orphan Medicine Assessment Report.
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: