EU/3/18/2025: Orphan designation for the treatment of methylmalonic acidaemia
Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles
Table of contents
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Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2023 on request of the Sponsor.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Withdrawn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. |
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June 2023 | The product was withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
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:
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: