This medicine is authorised for use in the European Union.


Defitelio is a medicine containing the active substance defibrotide. It is used to treat severe veno- occlusive disease (VOD) in patients undergoing haematopoietic (blood) stem-cell transplantation. VOD is a condition in which the veins in the liver become blocked, leading to liver dysfunction. Defitelio is used in adults and in children from one month of age.

VOD is rare, and Defitelio was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 July 2004. Further information on the orphan designation can be found here: medicine/Human medicines/Rare disease designation.

This EPAR was last updated on 31/07/2019

Authorisation details

Product details
Agency product number
Active substance
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
Hepatic Veno-Occlusive Disease
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Exceptional circumstancesExceptional circumstances

This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Gentium S.r.l.
Date of issue of marketing authorisation valid throughout the European Union
Contact address
Piazza XX Settembre 2
22079 Villa Guardia (Como)

Product information

11/07/2019 Defitelio - EMEA/H/C/002393 - S/0038


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Pharmacotherapeutic group

Antithrombotic agents

Therapeutic indication

Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.

It is indicated in adults and in adolescents, children and infants over 1 month of age.

Assessment history

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