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)

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.
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 Oxlumo 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 .

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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