EU/3/18/2052

About

On 31 July 2018, orphan designation (EU/3/18/2052) was granted by the European Commission to Dicerna EU Limited, United Kingdom, for synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues (also known as DCR-L1360) for the treatment of primary hyperoxaluria.

Key facts

Active substance
synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues
Disease / condition
Treatment of primary hyperoxaluria
Date of decision
31/07/2018
Outcome
Positive
Orphan decision number
EU/3/18/2052

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

Dicerna EU Limited
3rd Floor 11-12 St. James's Square
London SW1Y 4LB
United Kingdom
Tel. +44 (0)7771 808919
E-mail: http://dicerna.com/contact/

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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