Is the sodium salt of a complex mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA. It has been shown that defibrotide has protective effects on vascular endothelial cells, particularly those of small vessels. It has extensive beneficial pharmacological effects owing to its antithrombotic, anti-inflammatory and antiischemic properties.
Recent clinical trials of defibrotide indicate that this investigational drug may provide benefits both for the treatment of VOD and prophylaxis to prevent occurrence of VOD. Several different clinical trials indicate that defibrotide is well tolerated in patients.
Hepatic VOD is a potentially devastating complication of both allogeneic and autologous stem cell transplantation. Severe VOD usually occurs very early in the course of transplantation and the mortality rate of these cases is at least 90%.
The observed effects of Defibrotide in both preclinical and clinical trials have opened up new avenues of research for this drug in areas of unmet need such as cancer, inflammation, and thrombosis. Gentium's expert R&D team are dedicated to the investigation of Defibrotide's wide-ranging pharmacological activities.
Defibrotide is an investigational drug candidate and has not been approved by the U.S. Food and Drug Administration or any international regulatory agency.