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FDA Approves First Tx for Disease Linked to Stem Cell Transplant

Hepatic veno-occlusive disease is rare, but fatal

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WEDNESDAY, March 30, 2016 (HealthDay News) -- Defitelio (defibrotide sodium) has been approved by the U.S. Food and Drug Administration to treat a rare, but usually fatal, liver disease that affects some patients who have had hematopoietic stem cell transplantation.

Hepatic veno-occlusive disease (VOD) is fatal in as many as 80 percent of cases. It affects patients who have had hematopoietic stem cell transplantation and chemotherapy to treat cancers of the blood or bone marrow.

Defitelio was investigated in 528 people with hepatic VOD who participated in three clinical studies. The most common side effects included hypotension, diarrhea, vomiting, nausea, and epistaxis. More serious side effects could include hemorrhage and allergic reactions, the FDA said.

"The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT," Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in a statement.

Defitelio is marketed by Jazz Pharmaceuticals, based in Palo Alto, Calif.

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