Additional Relevant Details The FDA granted marketing approval for Defitelio (defibrotide sodium) for the treatment of adult and pediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following HSCT.


The FDA approval of Defitelio is supported by efficacy data from three clinical studies in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with Defitelio at the recommended 6.25 mg/kg every 6 hours; results are provided in the table below.

The safety of Defitelio to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT who were treated with Defitelio. The most common adverse reactions (incidence ≥10% and independent of causality) with Defitelio treatment were hypotension (low blood pressure), diarrhea, vomiting, nausea and epistaxis (nose bleeds).1  The most common serious adverse reactions (incidence ≥5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%).