EU/3/17/1959: Orphan designation for the treatment of tuberous sclerosis

Cannabidiol

Overview

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. 

The sponsorship was transferred to GW Pharma (International) B.V., the Netherlands, in April 2019.

Cannabidiol for treatment of tuberous sclerosis has been authorised in the EU as Epidyolex since 16 April 2021.

Key facts

Active substance
Cannabidiol
Intended use
Treatment of tuberous sclerosis
Orphan designation status
Positive
EU designation number
EU/3/17/1959
Date of designation
17/01/2018
Sponsor

GW Pharma (International) B.V. 
Databankweg 26
Amersfoort
Utrecht
3821 AL
Netherlands
Tel: +31 (0) 33 798 1015
E-mail: gwreg@gwpharm.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Cannabidiol at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

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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