Results from Phase 3 Trial of Defibrotide for the Treatment of Severe Veno-Occlusive Disease and Multi-Organ Failure Published Online

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that data from the phase 3 pivotal study of defibrotide were published online in BLOOD, the Journal of the American Society of Hematology (ASH).

The data demonstrated that defibrotide use in patients with hepatic veno-occlusive (VOD), also known as sinusoidal obstruction syndrome (SOS), with multi-organ failure (MOF) post-hematopoietic stem-cell transplantation (HSCT) was associated with a statistically significant improvement in Day +100 survival and in rate of complete response (CR) by Day +100, compared with rigorously selected historical controls. The manuscript can be found here:”

Based on the results of this pivotal phase 3 study, we believe defibrotide provides a promising treatment option for patients with this urgent unmet need,” said lead author and principal investigator, Paul G. Richardson, M.D., director of clinical research and clinical program leader at the Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute and the RJ Corman Professor of Medicine at Harvard Medical School, Boston MA. “Although HSCT has improved substantially over the last decade, hepatic VOD/SOS with MOF remains a very real and life-threatening complication post-HSCT, and for which there are no currently approved therapies.”

Defibrotide is an investigational agent in the United States (U.S.). A new drug application (NDA) is under review by the U.S. Food and Drug Administration (FDA). Hepatic VOD/SOS can be a life-threatening complication of HSCT. Hepatic VOD/SOS with MOF has been associated with 84% mortality rate.