EU/3/18/2033

About

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.

Key facts

Active substance
Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles
Disease / condition
Treatment of ornithine transcarbamylase deficiency
Date of decision
27/06/2018
Outcome
Positive
Orphan decision 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: +353 (1) 885 1710
E-mail: Info@realregulatory.com

Patients' organisations

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.

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