EU/3/18/2132

About

On 11 January 2019, orphan designation (EU/3/18/2132) was granted by the European Commission to Silence Therapeutics GmbH, Germany, for synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for the treatment of beta-thalassaemia intermedia and major.

Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Disease / condition
Treatment of beta-thalassaemia intermedia and major
Date of decision
11/01/2019
Outcome
Positive
Orphan decision number
EU/3/18/2132

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

Robert-Roessle-Strasse 10
13125 Berlin
Germany
Tel. +44 (0)1235 772 260
E-mail: st@s-cubed.co.uk

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;

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