FDA Approves Defibrotide for Hepatic VOD After Stem Cell Transplant

The FDA on Wednesday approved an investigational drug for the treatment of patients with hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction after a hematopoietic stem cell transplantation (HSCT).

The drug defibrotide sodium (Defitelio) was approved to treat VOD, also known as sinusoidal obstructive syndrome, in both adult and pediatric patients.

The approval was based on 2 prospective clinical trials and 1 expanded access study with 528 participants.

Every 6 hours, enrollees who had a diagnosis of hepatic VOD with multi-organ dysfunction after transplantation were administered 6.25 mg/kg of Defitelio intravenously until VOD was cleared.

The results of the study showed the Day +100 survival rates for study 1, 2, and 3 were 38% (95% CI: 29%, 48%), 44% (95% CI: 33%, 55%) and 45% (95% CI: 40%, 51%) respectively.

The Day +100 survival rates for hepatic VOD individuals with renal or pulmonary dysfunction who did not receive Defitelio, was 21% for supportive care and 31% for interventions.

The most common adverse events with incidence greater than or equal to 10% was diarrhea, epistaxis, hypotension, nausea, and vomiting. The major adverse events were hemorrhages and hypersensitivity.