EU/3/18/2026: Orphan designation for the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran)
Table of contents
Overview
On 25 May 2018, orphan designation (EU/3/18/2026) was granted by the European Commission to Alnylam UK Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (also known as ALN-TTRSC02) for the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis).
The medicinal product has been authorised in the EU as Amvuttra since 15 September 2022.
Key facts
Active substance |
synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran)
|
Intended use |
Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/18/2026
|
Date of designation |
25/05/2018
|
Sponsor |
Alnylam Netherlands B.V. |
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Amvuttra 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
Update history
Date | Update |
---|---|
November 2020 | The sponsor's address was updated. |
February 2019 | The sponsorship was transferred to Alnylam Netherlands B.V., The Netherlands. |
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: