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
Table of contents
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. |
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
Date | Update |
---|---|
June 2022 | The sponsorship was transferred to Moderna Biotech Spain S.L., Spain in June 2022. |
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: