Gentium's Defibrotide increases veno-occlusive disease survival rate, study says

By Angela McDaniels

Seattle, Dec. 7 - Gentium SpA said its investigational drug Defibrotide increased the 100-day survival rate of patients with veno-occlusive disease by 40% in a phase 2 trial, compared to the historic 100-day survival rate of 20%.

The 135-patient randomized, multi-center, dose-finding study investigated the use of Defibrotide as a treatment for veno-occlusive disease and multi-system organ failure as a complication of bone marrow and stem cell transplantation, the company said.

"It was particularly encouraging that in some of the most critically ill patients there was meaningful response to treatment. Our results showed response and long-term survival even in patients who were either on dialysis or ventilator-dependent at the start of Defibrotide therapy, which is really quite remarkable," lead investigator Paul G. Richardson of Harvard Medical School said in a company news release.

The findings will be presented on Dec. 12 at the 2005 Annual Meeting of the American Society of Hematology in Atlanta.

The company said it is about to begin a phase 3 study of Defibrotide for this indication in the United State.

Severe veno-occlusive disease is a potentially life-threatening condition with no approved treatment in which blood vessels in the liver become blocked, followed by liver and kidney failure, the company said. It is a side effect of cancer treatments such as high-dose chemotherapy and radiation.

Gentium is a biopharmaceutical company located in Villa Guardia, Italy, that develops drugs derived from DNA to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments.

Defibrotide is the company's lead product candidate in the United States and has been granted orphan drug status by the U.S. Food and Drug Administration to treat severe veno-occlusive disease and a fast track designation for the treatment of severe veno-occlusive disease in recipients of stem cell transplants.

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