EU/3/19/2224: Orphan designation for the treatment of CDKL5 deficiency disorder



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 medicinal product (for orphan indication in case of several orphan indications or type II variation) has been authorised in the EU as Ztalmy since 26 July 2023.

Key facts

Active substance
Intended use
Treatment of CDKL5 deficiency disorder
Orphan designation status
EU designation number
Date of designation

Marinus Pharmaceuticals Emerald Limited
10 Earlsfort Terrace
Dublin 2
D02 T380
Co. Dublin
Tel. +44 14848014670

Review of designation

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

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

Update history

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

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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