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爱尔兰爵士制药的去纤苷适应症

Author: Medicalhalo
Release time: 2025-10-19 11:44:20

(Defibrotide) is an oligonucleotide that can regulate platelet activity, inhibit thrombin release and activity, downregulate PAI-1 levels, have local anti-thrombosis and anti-inflammatory effects, promote fibrinolysis, and selectively act on small blood vessels. Defibrotide has proven effective in treating hepatic veno-occlusive disease (HVOD) with minimal adverse drug reactions, and has become a hot research topic in recent years.

On March 30, 2016, the U.S. FDA approved Defitelio® (defibrotide sodium) for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstructive syndrome (SOS), renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT). The launch of this drug provides another new option and opportunity for the treatment of patients with many indications.

Studies have found that defibrinoside can bind to plasmin, increase plasmin activity and promote fibrinolysis, but it cannot activate plasminogen to plasmin. Other studies have found that bacterial lipopolysaccharide (LPS) can induce the expression of TF in vascular endothelial cells, increase the level of plasminogen activation inhibitor-1 (PAI-1) and reduce the level of tissue plasminogen activator (t-PA), while defibrinoside can inhibit these effects of LPS, thus promoting fibrinolysis. Defibrinoside can also increase the expression of t-PA in resting endothelial cells, enhance fibrinolysis, and avoid fibrin deposition and vascular occlusion.

Irish Jazz Pharmaceuticals, the company to which Defibrotide belongs, is a biopharmaceutical company dedicated to developing and commercializing products that address unmet medical needs. It has a diversified portfolio of products and product candidates targeting the areas of sleep and hematology/oncology. It was first developed by Gentium Pharmaceuticals of Italy and was approved for marketing after Gentium Pharmaceuticals was acquired by Jazz Pharmaceuticals of the United States.

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