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FDA Approves Defibrotide for Hepatic VOD Following Stem Cell Transplantation

Dr. Richard Pazdur

Richard Pazdur, MD

The FDA has approved defibrotide sodium (Defitelio) as a treatment for severe hepatic veno-occlusive disease (VOD) with associated kidney or lung abnormalities following hematopoietic stem cell transplantation (HSCT), based on data from the collection of 3 studies.

Across the single-arm trials, two of which were prospective and one that was an expanded access study, 38% to 45% of patients were alive at 100 days following HSCT. The current 100-day survival rates with best supportive care following HSCT for patients with severe hepatic VOD are 21% to 31%, according to the FDA.

Severe hepatic VOD occurs in approximately 2% of patients treated with HSCT and has an 84% mortality rate. The approval for defibrotide followed a priority review by the FDA that was granted after a rolling submission that was allowed under a fast track designation. The PDUFA deadline was March 31, 2016. Click here to read the full article at

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