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.
Codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles
|Disease / condition||
Treatment of cystic fibrosis
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.