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)

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.
Antonio Vivaldistraat 150
1083 HP Amsterdam
Nord-Holland
Netherlands
Tel. +31 203697861
E-mail: info@alnylam.com

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 PDF icon orphan medicine assessment report

Update history

DateUpdate
November 2020The sponsor's address was updated.
February 2019The sponsorship was transferred to Alnylam Netherlands B.V., The Netherlands.

Documents related to this orphan designation evaluation

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:

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