On 27 June 2018, orphan designation (EU/3/18/2033) was granted by the European Commission to Real Regulatory Limited, Ireland, for codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles (also known as MRT5201) for the treatment of ornithine transcarbamylase deficiency.
EU/3/18/2033: Public summary of opinion on orphan designation: Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles for the treatment of ornithine transcarbamylase deficiency (PDF/129.18 KB)
First published: 21/08/2018
Last updated: 21/08/2018
Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles
|Disease / condition||
Treatment of ornithine transcarbamylase deficiency
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
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.