EU/3/18/2052: Orphan designation for the treatment of primary hyperoxaluria
synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
Table of contents
Overview
On 31 July 2018, orphan designation (EU/3/18/2052) was granted by the European Commission to Dicerna EU Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues (also known as DCR-L1360) for the treatment of primary hyperoxaluria.
The sponsorship was transferred to Dicerna Ireland Limited, Ireland in March 2019.
Key facts
| Active substance |
synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
|
| Intended use |
Treatment of primary hyperoxaluria
|
| Orphan designation status |
Positive
|
| EU designation number |
EU/3/18/2052
|
| Date of designation |
31/07/2018
|
| 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. |
| March 2019 | The sponsorship was transferred to Dicerna Ireland Limited, Ireland. |
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
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: