EU/3/14/1345: Orphan designation for the treatment of Cushing's syndrome



On 15 October 2014, orphan designation (EU/3/14/1345) was granted by the European Commission to Novartis Europharm Ltd, United Kingdom, for osilodrostat for the treatment of Cushing's syndrome.

The sponsorship was transferred to Novartis Europharm Limited, Ireland in May 2018.

Osilodrostat has been authorised in the EU as Isturisa since 9 January 2020.

The sponsorship was transferred to Recordati Rare Diseases, France in March 2020.

Key facts

Active substance
Intended use
Treatment of Cushing's syndrome
Orphan designation status
EU designation number
Date of designation

Recordati Rare Diseases   
Immeuble Le Wilson
70 Avenue Du General De Gaulle
92800 Puteaux
Tel. +33 1 4773 9527

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Isturisa 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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