Jazz Pharmaceuticals announced positive data from the Phase 3 pivotal study of defibrotide in patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with multi-organ failure (MOF) post-hematopoietic stem-cell transplantation (HSCT). Findings were published online in Blood, the Journal of the American Society of Hematology (ASH). 

The Phase 3 study evaluated the safety and efficacy of defibrotide 25 mg/kg/day in 102 patients with hepatic VOD/SOS with MOF compared to 32 historical controls identified from medical chart review by an independent medical review committee. The historical-control method offers a novel approach for Phase 3 studies of orphan diseases associated with high mortality, where a placebo control would be unethical. The primary endpoint was improvement in Day +100 post-HSCT survival.

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Study results showed that defibrotide was associated with a statistically significant improvement in Day +100 post-HSCT survival compared to historical controls, with an estimated difference of 23% (95% CI: 5.2%-40.8%; P=0.0109) between groups. A statistically significant improvement in rate of complete response (CR) by Day +100 post-HSCT was also seen, with an estimated difference adjusted for propensity score of 19% (95% CI: 3.5-34.6; P=0.0160) between groups. 

A New Drug Application (NDA) for defibrotide is currently under FDA review. It was previously granted Orphan Drug and Fast Track designations in 2003.

Defibrotide is being made available as an Investigational New Drug (IND) free of charge through an expanded access Treatment Protocol for the treatment of patients with hepatic VOD with evidence of MOD following HSCT.

For more information call (650) 496-377 or visit Jazzpharma.com