Defibrotide is a mixture of single-stranded oligonucleotides used in the treatment of severe hepatic veno-occlusive disease with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation. Defibrotide is the sodium salt of a mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA.
- Mechanism
- Curator reviewed
The drug appears to prevent the formation of blood clots and to help dissolve blood clots by increasing levels of prostaglandin I2, E2, and prostacyclin, altering platelet activity, increasing tissue plasminogen activator function, and decreasing activity of tissue plasminogen activator inhibitor. Prostaglandin I2 relaxes the smooth muscle of blood vessels and prevents platelets from adhering to each other. Prostaglandin E2 at certain concentrations also inhibits platelet aggregation. Moreover, the drug provides additional beneficial anti-inflammatory and antiischemic activities as recent sudies have shown. It is yet unclear, if the latter effects can be utilized clinically (e.g., treatment of ischemic stroke).
- Primary indication
- Indicated for the treatment of severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).Curator reviewed · 1 structured indication
- First approval
- Canada, 2017 · United States, 2016
- Also known as
- Defibrotide free acid · Defibrotide free acid (porcine mucosa) · Defibrotide free acid (porcine)
- Brand names
Resolves to
What you can answer from here — as of July 17, 2026
Which drugs share a target with Defibrotide, and which of those have an active Phase 3 trial?
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