EU/3/19/2141

Table of contents

About

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
Disease / condition
Treatment of haemophilia B
Date of decision
26/02/2019
Outcome
Positive
Orphan decision number
EU/3/19/2141

Sponsor's contact details

Fondazione Telethon
Via Varese 16B
00185 Roma
Italy
Tel. +39 0644 0151
E-mail: regulatory@telethon.it

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.

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