EU/3/16/1731: Orphan designation for the treatment of acute hepatic porphyria

Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran)

Overview

On 29 August 2016, orphan designation (EU/3/16/1731) was granted by the European Commission to Alnylam UK Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (also known as ALN-AS1) for the treatment of acute hepatic porphyria.

This medicine is now known as Givosiran.

The sponsorship was transferred to Alnylam Netherlands B.V., The Netherlands, in December 2018.

Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues has been authorised in the EU as Givlaari since 2 March 2020. 

The sponsor’s address was updated in November 2020.

Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues (givosiran)
Intended use
Treatment of acute hepatic porphyria
Orphan designation status
Positive
EU designation number
EU/3/16/1731
Date of designation
29/08/2016
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 Givlaari 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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