Last reviewed 2026-04-20 · How we verify

Defitelio (DEFIBROTIDE)

Defitelio works by inhibiting platelet aggregation and improving blood flow through the small blood vessels in the liver.

Defitelio (DEFIBROTIDE) is a small molecule drug originally developed by GENTIUM SRL and currently owned by Jazz Pharmaceuticals Inc. It was FDA-approved in 2016 for the treatment of veno-occlusive disease of the liver. The commercial status of Defitelio is patented, with no generic manufacturers available. Key safety considerations include the need for careful monitoring of patients due to potential bleeding risks. Defitelio is a treatment option for patients with severe liver disease.

At a glance

Mechanism of action

The mechanism of action of defibrotide sodium has not been fully elucidated. In vitro, defibrotide sodium enhances the enzymatic activity of plasmin to hydrolyze fibrin clots. Studies evaluating the pharmacological effects of defibrotide sodium on endothelial cells (ECs) were conducted primarily in the human microvascular endothelial cell line. In vitro, defibrotide sodium increased tissue plasminogen activator (t-PA) and thrombomodulin expression, and decreased von Willebrand factor (vWF) and plasminogen activator inhibitor-1 (PAI-1) expression, thereby reducing EC activation and increasing EC-mediated fibrinolysis. Defibrotide sodium protected ECs from damage caused by chemotherapy, tumor necrosis factor- (TNF-), serum starvation, and perfusion.

Approved indications

• Veno-occlusive disease of the liver

Common side effects

• Hypotension
• Diarrhea
• Vomiting
• Nausea
• Epistaxis
• Pulmonary alveolar hemorrhage
• Gastrointestinal hemorrhage
• Sepsis
• Graft versus host disease
• Lung infiltration
• Pneumonia
• Pulmonary hemorrhage

Key clinical trials

• Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (PHASE2)
• Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma (PHASE2)
• Defibrotide for the Treatment of Severe COVID-19 (PHASE2)
• Defibrotide Dose-escalation for SOS Post-HSCT (PHASE2)
• Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study
• Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome (PHASE2)
• Defibrotide in Children With High Risk Kawasaki Disease (PHASE2)
• Defibrotide as Prevention and Treatment of Respiratory Distress and Cytokine Release Syndrome of Covid 19. (PHASE2)

Patents

Primary sources

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