EU/3/14/1345: Orphan designation for the treatment of Cushing's syndrome
Table of contents
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.
Treatment of Cushing's syndrome
|Orphan designation status||
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Recordati Rare Diseases
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 orphan medicine assessment report .
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: