EU/3/04/212: Orphan designation for the treatment of hepatic veno-occlusive disease



On 29 July 2004, orphan designation (EU/3/04/212) was granted by the European Commission to, Gentium S.p.A, Italy, for defibrotide for the treatment of hepatic veno-occlusive disease (VOD).

In January 2016, Gentium S.p.A. changed name to Gentium S.r.l.

Update: Defibrotide has been authorised in the EU as Defitelio since 18 October 2013.

More information on Defitelio can be found in the European public assessment report (EPAR) on the Agency's website.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

Key facts

Active substance
Medicine name
Intended use
Treatment of hepatic veno-occlusive disease
Orphan designation status
EU designation number
Date of designation
Gentium S.r.l.
Piazza XX Settembre, 2
I-22079 Villa Guardia (CO)
Tel. +39 031 5373 200
Fax +39 031-5373 784

Review of designation

During its meeting of 3 to 4 September 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/04/212 for Defitelio (defibrotide) as an orphan medicinal product for the treatment of hepatic veno-occlusive disease. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Defitelio (defibrotide) for the treatment of hepatic veno-occlusive disease (PDF/78.56 KB)


    First published: 11/11/2013
    Last updated: 11/11/2013

  • Patients' organisations

    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.

    • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

    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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