On 17 January 2018, orphan designation (EU/3/17/1959) was granted by the European Commission to GW Research Ltd, United Kingdom, for cannabidiol for the treatment of tuberous sclerosis.
Cannabidiol for treatment of tuberous sclerosis has been authorised in the EU as Epidyolex since 16 April 2021.
Treatment of tuberous sclerosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|April 2019||The sponsorship was transferred to GW Pharma (International) B.V., the Netherlands.|
|June 2023||The sponsorship was transferred from GW Pharma (International) B.V., The Netherlands to Jazz Pharmaceuticals Ireland 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: