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
Table of contents
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 |
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
Date | Update |
---|---|
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. |
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: