On 27 June 2016, orphan designation (EU/3/16/1684) was granted by the European Commission to Alexion Europe SAS, France, for modified mRNA encoding the UGT1A1 protein for the treatment of Crigler-Najjar syndrome.
The sponsorship was transferred to Pharma Gateway AB - Sweden, in May 2018.
Modified mRNA encoding the UGT1A1 protein
Treatment of Crigler-Najjar syndrome
|Orphan designation status||
|EU designation number||
|Date of designation||
|September 2022||The sponsorship was transferred from Pharma Gateway AB, Sweden to Moderna Biotech Spain S.L., Spain.|
|May 2018||The sponsorship was transferred to Pharma Gateway AB - Sweden.|
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: