Jazz Pharmaceuticals Stops Enrollment in Phase 3 Study Evaluating Defibrotide for the Prevention of Veno-Occlusive Disease

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[{"type":"text","content":"DUBLIN, April 29, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that enrollment in the Phase 3 clinical study of defibrotide for the prevention of hepatic veno-occlusive disease (VOD) post-hematopoietic stem-cell transplantation (HSCT) in high-risk or very high-risk patients is being discontinued. Enrollment in the study was stopped early based on the recommendation from an Independent Data Monitoring Committee (IDMC), which concluded it would be highly unlikely to reach statistical significance for the primary endpoint of VOD-free survival at Day +30 post-HSCT in the final analysis if the study were to complete enrollment. \nThe IDMC recommendation was made after completion of a pre-planned interim analysis of the first 280 patients and where the study met the protocol-specified futility criteria. The study was conducted as part of a post-marketing requirement related to safety. The IDMC conducted multiple periodic safety analyses during the study and reported no new safety concerns.\n\"We are disappointed with this outcome, but wish to thank the patients and physicians involved in the program for their participation in the study,\" said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. \"While enrollment has been discontinued, the trial will run to completion through the follow-up of the more than 370 patients enrolled. VOD is a rapidly progressive and devastating condition that can develop following stem-cell transplantation. As the only approved treatment with demonstrated efficacy based on survival at 100 days after HSCT, defibrotide is an important medicine for this patient population.\"\nJazz has notified relevant health authorities and study investigators of the decision to discontinue new enrollment to the study. Patients enrolled in the study will be allowed to continue participation if they and the investigator determine this would be in the patient's best interest. Patients will continue to be followed per protocol. The full data will be submitted to a future medical meeting for presentation. \nThis Phase 3 trial was not designed to further evaluate defibrotide for the treatment of VOD and does not impact the approved indication or other ongoing clinical studies. Defitelio® (defibrotide sodium) remains the only treatment approved...

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