On 26 February 2019, orphan designation (EU/3/19/2141) was granted by the European Commission to Fondazione Telethon, Italy, for lentiviral vector encoding human coagulation factor IX for treatment of haemophilia B.
Lentiviral vector encoding human coagulation factor IX
|Disease / condition||
Treatment of haemophilia B
|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: