EU/3/05/296: Orphan designation for the treatment of congenital adrenal hyperplasia
Table of contents
On 27 July 2005, orphan designation (EU/3/05/296) was granted by the European Commission to Professor Richard JM Ross, United Kingdom, for hydrocortisone (modified release tablet) for the treatment of congenital adrenal hyperplasia.
The sponsorship was transferred to Phoqus Pharmaceuticals Limited, United Kingdom, in December 2006, to Diurnal Limited, United Kingdom, in February 2009 and subsequently to Diurnal Europe B.V., Netherlands, in February 2019.
On 26 February 2020 the active substance was corrected from hydrocortisone (modified release tablet) to hydrocortisone.
Please note that this product (marketed as marketed as Efmody) was withdrawn from the Union Register of orphan medicinal products in April 2021 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.
Treatment of congenital adrenal hyperplasia
|Orphan designation status||
|EU designation number||
|Date of designation||
Diurnal Europe B.V.
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of hydrocortisone at the time of marketing authorisation.
The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the withdrawal assessment report – orphan maintenance.
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: