EU/3/16/1637: Orphan designation for the treatment of primary hyperoxaluria
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran)
Table of contents
Overview
This medicine is now known as lumasiran.
On 21 March 2016, orphan designation (EU/3/16/1637) was granted by the European Commission to Alnylam UK Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for the treatment of primary hyperoxaluria.
The sponsorship was transferred to Alnylam Netherlands B.V. the Netherlands, in February 2019.
The sponsor’s address was updated in November 2020.
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues has been authorised in the EU as Oxlumo since 19 November 2020.
Key facts
Active substance |
Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran)
|
Intended use |
Treatment of primary hyperoxaluria
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/16/1637
|
Date of designation |
21/03/2016
|
Sponsor |
Alnylam Netherlands B.V. |
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Oxlumo at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the
orphan medicine assessment report
.
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: