FDA Approves Defibrotide for Complication of Stem Cell Transplant


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On March 30, 2016, the U.S. Food and Drug Administration (FDA) approved defibrotide sodium (Defitelio) for the treatment of adult and pediatric patients with hepatic veno-occlusive disease, also known as sinusoidal obstructive syndrome, with renal or pulmonary dysfunction following hematopoietic stem cell transplantation. This is the first FDA-approved therapy for treatment of severe hepatic veno-occlusive disease.

Clinical Trials

The efficacy of defibrotide sodium was investigated in 528 patients treated on three studies: two prospective clinical trials (study 1 and study 2) and an expanded access study (study 3). The patients enrolled had a diagnosis of hepatic veno-occlusive disease with multiorgan dysfunction after transplantation. They received defibrotide sodium at 6.25 mg/kg intravenously every 6 hours until resolution of veno-occlusive disease.

The approval was based on survival at 100 days after hematopoietic stem-cell transplantation. The day +100 survival rates for study 1, study 2, and study 3 were 38% (95% confidence interval [CI] = 29%–48%); 44% (95% CI = 33%–55%); and 45% (95% CI = 40%–51%), respectively. Based on published reports and analyses of patient-level data, the day +100 survival rates were 21% to 31% for patients with hepatic veno-occlusive disease with renal or pulmonary dysfunction who received supportive care or interventions other than defibrotide sodium.

For more information visit the Office of Hematology and Oncology Products at www.FDA.gov. ■



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