DEFITELIO
Defitelio (defibrotide sodium) is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS). This therapy is specifically used for patients who develop renal or pulmonary dysfunction following a hematopoietic stem-cell transplantation (HSCT). It serves as a specialized treatment for these severe complications in both adult and pediatric populations.
How DEFITELIO Works
Defibrotide sodium protects endothelial cells and enhances the breakdown of blood clots, though its precise mechanism is not fully elucidated. In vitro studies show the drug increases the expression of tissue plasminogen activator and thrombomodulin while decreasing factors that promote cell activation and clotting. These actions enhance fibrinolysis and protect endothelial cells from damage caused by chemotherapy and inflammatory cytokines.
Details
- Status
- Prescription
- First Approved
- 2016-03-30
- Routes
- INTRAVENOUS
- Dosage Forms
- SOLUTION
DEFITELIO Approval History
What DEFITELIO Treats
4 indicationsDEFITELIO is approved for 4 conditions since its original approval in 2016. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hepatic Veno-Occlusive Disease
- Sinusoidal Obstruction Syndrome
- Renal Dysfunction
- Pulmonary Dysfunction
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Active Pipeline
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Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
DEFITELIO FDA Label Details
ProIndications & Usage
FDA Label (PDF)DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT). DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.