On 14 December 2015, orphan designation (EU/3/15/1585) was granted by the European Commission to Rare Partners srl Impresa Sociale, Italy, for sirolimus for the treatment of beta-thalassaemia intermedia and major.
The sponsor’s address was updated in May 2020.
|Disease / condition||
Treatment of beta thalassaemia intermedia and major
|Date of first decision||
|EU designation number||
Review of designation
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.