On 29 December 2000, orphan designation (EU/3/00/012) was granted by the European Commission to Swedish Orphan AB, Sweden, for nitisinone for the treatment of tyrosinaemia type 1.
Swedish Orphan AB changed name to Swedish Orphan International AB in January 2003 and then to Swedish Orphan Biovitrum International AB, in August 2013.
Nitisinone has been authorised in the EU as Orfadin since 21 February 2005.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Treatment of tyrosinaemia type 1
|Orphan designation status||
|EU designation number||
|Date of designation||
Documents related to this orphan designation evaluation
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
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: