EU/3/17/1829: Orphan designation for the treatment of Dravet syndrome

26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound

Overview

On 27 February 2017, orphan designation (EU/3/17/1829) was granted by the European Commission to EirGen Pharma Limited, Ireland, for 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound (also known as CUR-1916) for the treatment of Dravet syndrome.

Key facts

Active substance
26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound
Intended use
Treatment of Dravet syndrome
Orphan designation status
Positive
EU designation number
EU/3/17/1829
Date of designation
27/02/2017
Sponsor

S-Cubed Pharmaceutical Services ApS
Baneledet 2
3600 Frederikssund
Hovedstaden
Denmark
E-mail: info@s-cubed-global.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
April 2022The sponsorship was transferred to S-cubed Pharmaceutical Services ApS, Denmark in April 2022.
June 2023The sponsor’s address was updated in June 2023.

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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