EU/3/19/2235: Orphan designation for the treatment of congenital alpha-1 antitrypsin deficiency

Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues

Overview

On 16 December 2019, orphan designation EU/3/19/2235 was granted by the European Commission to Dicerna Ireland Limited, Ireland, for synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues (also known as DCR S1459) for the treatment of congenital alpha-1 antitrypsin deficiency.

Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
Intended use
Treatment of congenital alpha-1 antitrypsin deficiency
Orphan designation status
Positive
EU designation number
EU/3/19/2235
Date of designation
16/12/2019
Sponsor

Dicerna Ireland Limited
10 Earlsfort Terrace
Dublin 2
Co. Dublin
D02 T380
Ireland
E-mail: contact@dicerna.com

Review of designation

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

Update history

DateUpdate
February 2023The sponsor's address was updated.

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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