EU/3/19/2141: Orphan designation for the treatment of haemophilia B

Lentiviral vector encoding human coagulation factor IX

Table of contents


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.

Key facts

Active substance
Lentiviral vector encoding human coagulation factor IX
Intended use
Treatment of haemophilia B
Orphan designation status
EU designation number
Date of designation

Genespire S.r.l.
Via Vincenzo Gioberti 8
20123 Milan

Update history

December 2022The sponsorship was transferred from Fondazione Telethon Ets, Italy to Genespire S.r.l., Italy.
November 2022The sponsor's name was changed from Fondazione Telethon to Fondazione Telethon Ets.

Documents related to this orphan designation evaluation

Patients' organisations

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:

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