EU/3/19/2150: Orphan designation for the treatment of cystic fibrosis
Codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles
Table of contents
Overview
On 1 April 2019, orphan designation (EU/3/19/2150) was granted by the European Commission to Real Regulatory Limited, Ireland, for codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles (also known as MRT5005) for the treatment of cystic fibrosis.
The sponsorship was transferred to Sanofi-Aventis Group, France in February 2022.
Key facts
Active substance |
Codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles
|
Intended use |
Treatment of cystic fibrosis
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/19/2150
|
Date of designation |
01/04/2019
|
Sponsor |
Sanofi-Aventis Groupe |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
This medicine is now known as pomulmeran.
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: