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.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in April 2021 on request of the Sponsor.
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/220.94 KB)
First published: 21/08/2018
Last updated: 21/08/2018
Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles
Treatment of ornithine transcarbamylase deficiency
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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.