Defibrotide Highlighted in Presentation at the 51st Meeting of the German Society for Thrombosis and Hemostasis
Defibrotide is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration to prevent and to treat severe veno-occlusive disease (VOD) and Fast Track designation for the treatment of severe VOD in recipients of stem cell transplants. Defibrotide is currently in a U.S. Phase III trial to treat severe VOD and a European Phase II/III trial to prevent VOD in pediatric patients.
In his lecture before a primarily physician-based audience, Professor Holler is scheduled to give an overview on pre-clinical and clinical data obtained about Defibrotide. In addition to pharmacological and pharmacokinetic information, he will describe early animal experiments on the protective role of Defibrotide in ischemic-reperfusion injury, as well as in vitro experiments dealing with the anti-apoptotic activity of Defibrotide in endothelial cells, along with data on new biological effects of the drug. Professor Holler will also focus on clinical experiences in the treatment of VOD after allogeneic stem cell transplantation. Finally, he will present information about ongoing multi-center trials using Defibrotide for the prevention of VOD and transplant-associated microangiopathy.
Commenting on the upcoming lecture, Laura Ferro, M.D., president and chief executive officer of Gentium, said, "We are very pleased to have Professor Holler lecture on Defibrotide as further knowledge of its mechanism of action leads to a better understanding of its clinical potential in a number of important medical indications of unmet need. In addition, such information appears to corroborate scientifically what we have demonstrated in our clinical trials and reinforces confidence in our clinical development strategy."
Veno-occlusive disease (VOD) is a potentially life-threatening condition. Certain high-dose chemotherapy and radiation therapies and stem cell transplantation (SCT) can damage cells of the blood vessels and result in VOD, a blockage of the small veins of the liver that can lead to liver failure and the failure of other organs (severe VOD). SCT is a frequently used treatment following high-dose chemotherapy and radiation therapy. The International Bone Marrow Transplant Registry estimated that in 2002 approximately 45,000 people received blood and bone marrow transplants, which are types of SCT. Based on the Company's review of more than 200 published papers, it believes that approximately 20% of patients who undergo SCT develop VOD, approximately one-third of those who develop VOD progress to severe VOD and approximately 80% of severe VOD patients die within 100 days of the SCT. The Company believes that there are no approved therapies to treat or prevent VOD in the U.S. or the EU.
Gentium S.p.A. is a biopharmaceutical company focused on the research, discovery and development of drugs derived from DNA extracted from natural sources, and drugs that are synthetic derivatives, to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate in the U.S., is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration to prevent and to treat VOD and Fast Track designation for the treatment of severe VOD in recipients of stem cell transplants.
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Posted: February 2007