EU/3/16/1731

About

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. 

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)
Disease / condition
Treatment of acute hepatic porphyria
Date of first decision
29/08/2016
Outcome
Positive
EU designation number
EU/3/16/1731

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 iconorphan medicine assessment report .

Sponsor's contact details

Alnylam Netherlands B.V.
Strawinskylaan 3051
1077ZX
Amsterdam
The Netherlands
Tel. + 31 20 369 0716
E-mail: info@alnylam.com

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.

How useful was this page?

Add your rating