On 9 January 2020, orphan designation EU/3/19/2245 was granted by the European Commission to TMC Pharma (EU) Limited, Ireland, for setmelanotide for the treatment of Alström syndrome.
The sponsorship was transferred to Rhythm Pharmaceuticals Limited, Ireland, in November 2020.
|Disease / condition||
Treatment of Alström syndrome
|Date of first decision||
|EU designation number||
Sponsor's contact details
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.