EU/3/18/2025: Orphan designation for the treatment of methylmalonic acidaemia

Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles

Overview

Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2023 on request of the Sponsor.

On 25 May 2018, orphan designation (EU/3/18/2025) was granted by the European Commission to Pharma Gateway AB, Sweden, for modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles (also known as mRNA-3704) for the treatment of methylmalonic acidaemia.

Key facts

Active substance
Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles
Intended use
Treatment of methylmalonic acidaemia
Orphan designation status
Withdrawn
EU designation number
EU/3/18/2025
Date of designation
25/05/2018
Sponsor

Moderna Biotech Spain S.L.
Calle Del Principe De Vergara 132 Plt 12
28002 Madrid
Spain
Email: Luke.MirceaWillats@modernatx.com

Review of designation

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

Update history

DateUpdate
June 2023The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.
June 2022The sponsorship was transferred to Moderna Biotech Spain S.L., Spain in June 2022.

Documents related to this orphan designation evaluation

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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