Global InformationDefibrotide information
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| Trade names | Defitelio |
| Other names | STA-1474, JZP-381, defibrotide sodium (JAN JP), defibrotide sodium (USAN US) |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | Intravenous |
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| Bioavailability | 58 - 70% by mouth (i.v. and i.m. = 100%) |
| Elimination half-life | < 2 hours[5] |
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Defibrotide, sold under the brand name 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 the European Union. It works by protecting the cells lining blood vessels in the liver and preventing blood clotting; the way it does this is not well understood.[5][4][6]
The most common side effects include abnormally low blood pressure (hypotension), diarrhea, vomiting, nausea and nosebleeds (epistaxis). Serious potential side effects that were identified include bleeding (hemorrhage) and allergic reactions. Defibrotide should not be used in people who are having bleeding complications or who are taking blood thinners or other medicines that reduce the body's ability to form clots.[7][6] Use of the drug is generally limited by a strong risk of life-threatening bleeding in the brain, eyes, lungs, gastrointestinal tract, urinary tract, and nose. Some people have hypersensitivity reactions.[4]
Defibrotide was approved for medical use in the European Union in October 2013, in the United States in March 2016, and in Australia in July 2020.[8][6][1] Defibrotide is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition.[7]
- ^ a b c "Defitelio Australian Prescription Medicine Decision Summary". Therapeutic Goods Administration (TGA). 31 July 2020. Retrieved 16 August 2020.
- ^ "Blood health (non-cancer)". Health Canada. 11 May 2018. Retrieved 13 April 2024.
- ^ "Regulatory Decision Summary - Defitelio". Drug and Health Product Register. 23 October 2014. Retrieved 13 April 2024.
- ^ a b c "Defitelio 80 mg/mL concentrate for solution for infusion - Summary of Product Characteristics". UK Electronic Medicines Compendium. 26 May 2016. Retrieved 20 July 2017.
- ^ a b c "Defitelio- defibrotide sodium injection, solution". DailyMed. 30 March 2016. Retrieved 16 August 2020.
- ^ a b c d "Defitelio EPAR". European Medicines Agency (EMA). Retrieved 16 August 2020.
- ^ a b Cite error: The named reference
FDA PRwas invoked but never defined (see the help page). - ^ "Defitelio Injection". U.S. Food and Drug Administration (FDA). 10 May 2016. Retrieved 16 August 2020.