EU/3/18/2033:

Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles

Overview

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Please note that this product was withdrawn from the Union Register of orphan medicinal products in April 2021 on request of the Sponsor.

Key facts

Active substance
Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles
Intented use
Treatment of ornithine transcarbamylase deficiency
Date of designation
27/06/2018
Orphan designation status
Withdrawn
EU designation number
EU/3/18/2033

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Real Regulatory Limited
Unit 9A, Plato Business Park
Damastown
Dublin 15 D15 PA4C
Ireland
Tel: +35318851710
E-mail: Info@realregulatory.com

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.

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