EU/3/19/2156: Orphan designation for the treatment of propionic acidaemia

Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle

Overview

On 24 April 2019, orphan designation (EU/3/19/2156) was granted by the European Commission to Pharma Gateway AB, Sweden, for modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle (also known as mRNA-3927) for the treatment of propionic acidaemia.

Key facts

Active substance
Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle
Intended use
Treatment of propionic acidaemia
Orphan designation status
Positive
EU designation number
EU/3/19/2156
Date of designation
24/04/2019
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 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:

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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