On 13 November 2019, orphan designation EU/3/19/2224 was granted by the European Commission to Pharma Gateway AB, Sweden, for ganaxolone for the treatment of CDKL5 deficiency disorder.
Treatment of CDKL5 deficiency disorder
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|June 2021||The sponsorship was transferred to Marinus Pharmaceuticals Emerald Limited, Ireland.|
Documents related to this orphan designation evaluation
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: