On 22 August 2014, orphan designation (EU/3/14/1309) was granted by the European Commission to NDA Group AB, Sweden, for 17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione for the treatment of Duchenne muscular dystrophy.
The medicine is now known as vamorolone.
Treatment of Duchenne muscular dystrophy
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|May 2015||The sponsorship was transferred to ReveraGen BioPharma Limited, United Kingdom|
|January 2019||The sponsorship was transferred to Pharma Gateway AB, Sweden|
|September 2022||The sponsorship was transferred from Insidereg Limited, Ireland to Pharma Gateway AB, Sweden in September 2022.|
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: