EU/3/19/2224:

Ganaxolone

Overview

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.

The sponsorship was transferred to Marinus Pharmaceuticals Emerald Limited, Ireland, in June 2021.

Key facts

Active substance
Ganaxolone
Intented use
Treatment of CDKL5 deficiency disorder
Date of designation
13/11/2019
Orphan designation status
Positive
EU designation number
EU/3/19/2224

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product it it is approved for marketing authorisation.

Sponsor's contact details

Marinus Pharmaceuticals Emerald Limited
10 Earlsfort Terrace
Dublin 2
D02 T380
Co. Dublin
Ireland
Tel. +44 14848014670
E-mail: Medicalaffairs@marinuspharma.com

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.

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