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)
Table of contents
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. |
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
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: