|AHFS/Drugs.com||International Drug Names|
|Bioavailability||58 - 70% by mouth (i.v. and i.m. = 100%)|
|Elimination half-life||< 2 hours|
|(what is this?)|
Defibrotide, sold under the brandname Defitelio, is a mixture of single-stranded oligonucleotides that is purified from the intestinal mucosa of pigs. It is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant, with different limitations in the US and Europe. It works by protecting the cells lining bloods vessels and preventing blood clotting; the way it does this is not well understood.
There is a strong risk of bleeding in the brain, eyes, lungs, gastrointestinal tract, urinary tract, and nose. Some people have hypersensitivity reactions.
It was approved in Europe in 2013 and in the US in 2016.
Defibrotide is used to treat veno-occlusive disease of the liver of people having had a bone marrow transplant, with different limitations in the US and Europe. As of 2016, however, randomized placebo controlled trials have not been done.
It is administered by intravenous infusion in a doctor's office or clinic.
Use of defibrotide for people who are already taking anticoagulants is dangerous and use of other drugs that affect platelet aggregation, like NSAIDs, should be done with care. Defibrotide should not be given to people who have a difficult time maintaining a steady blood pressure.
Common adverse effects, occurring in between 1 and 10% of people, included impaired blood clotting, vomiting, low blood pressure, bleeding in the brain, eyes, lungs, stomach or intestines, in the urine, and at catheterization sites.
Defibrotide's mechanism of action is poorly understood. In vitro studies have shown that it protects the endothelium lining blood vessels from damage by fludarabine, a chemotherapy drug, and from a few other insults like serum starvation. It also appears to increase t-PA function and decrease plasminogen activator inhibitor-1 activity.
Society and culture
Defibrotide was approved in Europe for use in treating veno-occlusive disease of the liver of people having had a bone marrow transplant in 2013; Gentium had developed it. At the end of that year, Jazz Pharmaceuticals acquired Gentium. In March 2016 the US FDA approved it for a similar use.
- "Defibrotide sodium label" (PDF). FDA. March 2016. Retrieved 20 July 2017. For label updates see FDA index page for NDA 208114
- "Defitelio 80 mg/mL concentrate for solution for infusion - Summary of Product Characteristics". UK Electronic Medicines Compendium. 26 May 2016. Retrieved 20 July 2017.
- Dalle, JH; Giralt, SA (March 2016). "Hepatic Veno-Occlusive Disease after Hematopoietic Stem Cell Transplantation: Risk Factors and Stratification, Prophylaxis, and Treatment". Biology of Blood and Marrow Transplantation. 22 (3): 400–9. doi:10.1016/j.bbmt.2015.09.024. PMID 26431626.
- "Jazz Pharma Acquiring Gentium for $1B". GEN Genetic Engineering & Biotechnology News. December 20, 2013.
- "Press Announcements - FDA approves first treatment for rare disease in patients who receive stem cell transplant from blood or bone marrow". FDA. March 30, 2016.