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: ema.europa.eu/Find medicine/Human medicines/Rare disease designation.
Defitelio : EPAR - Medicine overview (PDF/81.94 KB) (updated)
First published: 25/10/2013
Last updated: 17/05/2019
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Hepatic Veno-Occlusive Disease
|Anatomical therapeutic chemical (ATC) code||
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.
|Date of issue of marketing authorisation valid throughout the European Union||
26/07/2018 Defitelio - EMEA/H/C/002393 - R/0032
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
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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.