# Defibrotide

Source: https://onco.cc/drugs/defibrotide/  
OnCo record `defibrotide` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Defibrotide (Defitelio) is the only approved treatment for hepatic veno-occlusive disease, a life-threatening liver complication of high-dose chemotherapy before stem cell transplant, in patients whose kidneys or lungs are also failing.

## Summary

Defibrotide was approved by the FDA in March 2016 for adults and children with hepatic veno-occlusive disease (sinusoidal obstruction syndrome) with renal or pulmonary dysfunction after haematopoietic stem cell transplantation, on a phase 3 trial against historical controls in which survival at day 100 was 38% versus 25%, supported by an expanded-access programme of several hundred patients; the EU authorised Defitelio in 2013 for severe VOD after HSCT in patients over one month old. A subsequent phase 3 prophylaxis trial in high-risk patients (HARMONY) did not meet its primary endpoint, so use remains therapeutic. Bleeding is the main risk and it is contraindicated with systemic anticoagulants or thrombolytics. Marketed by Jazz Pharmaceuticals.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-24
- Tags: nci-list; supportive
- Brand: Defitelio
- Modality: Polydisperse oligonucleotide mixture (endothelial protectant)
- Mechanism: Mixture of single-stranded oligonucleotides from porcine intestinal mucosa that enhances plasmin-mediated fibrinolysis, reduces endothelial activation and protects sinusoidal endothelial cells; the mechanism is not fully elucidated.
- Approvals: US 2016: Hepatic veno-occlusive disease with renal or pulmonary dysfunction after HSCT, adults and children; EU 2013: Severe hepatic veno-occlusive disease after HSCT in patients over one month (Defitelio)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Defibrotide
- Study 2005-01 (Blood 2016): https://doi.org/10.1182/blood-2015-10-676924
- US label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2c3db989-d7ad-41ed-9ebf-698dcf6c24ec
- NCI drug page: https://www.cancer.gov/about-cancer/treatment/drugs/defibrotide-sodium
- EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/defitelio

## Connected records

- technologies: [Allogeneic stem cell transplantation](https://onco.cc/technologies/allogeneic-hsct/), [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/)
- companies: [Jazz Pharmaceuticals](https://onco.cc/companies/jazz/)
- trials: [Study 2005-01](https://onco.cc/trials/study-2005-01/)
- key papers: [Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure](https://onco.cc/key-papers/paper-defibrotide-2005-01-blood-2016/)

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