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FDA Says “Yes” to Defibrotide (Defitelio) for Hepatic Veno-Occlusive Disease Following Stem-Cell Transplantation

First approved therapy for patients with rare, potentially fatal complication

The FDA has granted marketing approval for defibrotide sodium (Defitelio, Jazz Pharmaceuticals) 80 mg/mL for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

The approval of defibrotide was supported by efficacy data from three clinical trials in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with the drug at the recommended dosage of 6.25 mg/kg every six hours. In a phase 3 prospective study, the survival rate at day 100 after HSCT was 38% in 102 patients; in a phase 2 prospective study, the survival rate was 44% in 75 patients; and in an expanded-access study, the survival rate was 45% in 351 patients.

The safety of defibrotide was based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT. The most common adverse events associated with defibrotide included hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse events were hypotension (11%) and pulmonary alveolar hemorrhage (7%).

Source: Jazz Pharmaceuticals; March 30, 2016.

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