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.
26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound
Treatment of Dravet syndrome
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|April 2022||The sponsorship was transferred to S-cubed Pharmaceutical Services ApS, Denmark in April 2022.|
|June 2023||The sponsor’s address was updated in June 2023.|
Documents related to this orphan designation evaluation
EU/3/17/1829: Public summary of opinion on orphan designation: 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound for the treatment of Dravet syndrome (PDF/131.21 KB)
First published: 31/03/2017
Last updated: 31/03/2017
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: