EU/3/19/2235: Orphan designation for the treatment of congenital alpha-1 antitrypsin deficiency
Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
Table of contents
Overview
On 16 December 2019, orphan designation EU/3/19/2235 was granted by the European Commission to Dicerna Ireland Limited, Ireland, for synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues (also known as DCR S1459) for the treatment of congenital alpha-1 antitrypsin deficiency.
Key facts
Active substance |
Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
|
Intended use |
Treatment of congenital alpha-1 antitrypsin deficiency
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/19/2235
|
Date of designation |
16/12/2019
|
Sponsor |
Dicerna Ireland Limited |
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 |
---|---|
February 2023 | The sponsor's address was updated. |
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: