Gentium S.p.A. We are a biopharmaceutical company, headquartered in Villa Guardia (Como) - Italy, focused on research, development and manufacture of active ingredients derived from natural sources as potential therapeutic agents. Our mission is to discover and provide new therapies to care-givers for diseases that currently have few or no treatment options with the aim to improve the lives of those faced with rare diseases with high unmet medical needs. Defibrotide, an investigational drug based on single-stranded DNA extracted from pig intestines, is our most advanced product candidate. In the U.S., Defibrotide has been granted “orphan” status by the Food and Drug Administration (FDA) for the treatment of hepatic veno-occlusive disease. In The EU, the European Medicines Agency (EMA) has granted Defibrotide orphan status for the prevention of hepatic veno-occlusive disease. Currently, Defibrotide is under development for the treatment and prevention of a rare disease called hepatic veno-occlusive disease, or VOD, a condition in which some of the veins in the liver are blocked as a result of cancer treatments, such as chemotherapy or radiation treatments, that are given prior to stem cell transplant. Severe VOD is the most extreme form of VOD and is associated with multiple-organ failure and high rates of morbidity and mortality. Currently, there are no approved treatments for VOD by either the U.S. FDA or EMA. Two pivotal Phase II/III clinical trials with defibrotide for the treatment and prevention of VOD have been completed, a Phase III trial for treatment of severe VOD in the U.S., Canada and Israel and Phase II/III pediatric trial in Europe for the prevention of VOD. In the absence of approved treatments for VOD, defibrotide has been made available as an investigational new drug, through a Treatment IND Protocol for patients with VOD (in the U.S.) and on a named patient basis in Europe.