TheraRadar
Data updated: May 26, 2026

DEFITELIO (defibrotide sodium)

Genetic Support
First-in-Class Orphan Drug Priority Review Fast Track
Hepatology Approved 2016-03-30

DEFITELIO is indicated for the treatment of Hepatic Veno-Occlusive Disease; Sinusoidal Obstruction Syndrome; Renal Dysfunction; Pulmonary Dysfunction.

Source: FDA Label • JAZZ PHARMS INC

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.

1
Indication
--
Phase 3 Trials
1
Priority Reviews
10
Years on Market

Details

Status
Prescription
First Approved
2016-03-30
Patent Cliff
2032

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Routes
INTRAVENOUS
Dosage Forms
SOLUTION

Companies

Active Ingredient: DEFIBROTIDE SODIUM

DEFITELIO Approval History

2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
1 FDA actions from 2016 to 2016
Mar 2016 ORIGINAL Priority
New Drug · Type 1 - New Molecular Entity

What DEFITELIO Treats

4 indications

DEFITELIO 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
Source: FDA Label

DEFITELIO Target & Pathway

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Target

TNF (Tumor Necrosis Factor) Cytokine

A pro-inflammatory cytokine that plays a central role in immune responses and inflammation. Excess TNF contributes to autoimmune diseases like rheumatoid arthritis, psoriasis, and inflammatory bowel disease. Blocking TNF reduces inflammation and prevents tissue damage.

DEFITELIO Competitive Set

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Three rings of competition based on shared molecular targets and treated indications.

MoA expansion candidates

2

Same target(s), different indications — where else is this mechanism being explored?

Indication competitors

1

Same indication, different mechanism — what else might this patient receive?

Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

Drugs Similar to DEFITELIO

FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.

HYDROCHLOROTHIAZIDE
HYDROCHLOROTHIAZIDE
1 shared
WATSON LABS
Shared indications:
Renal Dysfunction
🔬

Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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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

Indications & Usage

FDA Label (PDF)

DEFITELIO is indicated for the treatment of Hepatic Veno-Occlusive Disease; Sinusoidal Obstruction Syndrome; Renal Dysfunction; Pulmonary Dysfunction.

View full patent landscape →
3 OB patents · 1 families · 50 international docs across 17 countries

DEFITELIO Patents & Exclusivity

Latest Patent: Jun 2032

Patents (5 active)

US11236328 Expires Jun 22, 2032
US11085043 Expires Jun 22, 2032
US12529052 Expires Jun 22, 2032
US11746348 Expires Jun 22, 2032
US12534722 Expires Jun 22, 2032
Source: FDA Orange Book

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Revenue Insights

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  • Historical trend analysis

Patent Timeline

  • Cliff: 2032
  • 5 active patents

Trial Analysis

  • Clinical trial tracking
  • Development stage analysis

Competitive Landscape

  • 1 similar drugs
  • Same target/indication analysis
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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.