EU/3/16/1637

About

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.

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)
Disease / condition
Treatment of primary hyperoxaluria
Date of first decision
21/03/2016
Outcome
Positive
EU designation number
EU/3/16/1637

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Alnylam Netherlands B.V. 
Strawinskylaan 3051
1077ZX Amsterdam
The Netherlands
Tel. +31 6152 12490
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