Jazz Pharmaceuticals to present defibrotide results for hepatic VOD at BMT Tandem meetings

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced today that researchers will present data on the use of defibrotide, an investigational medicine being studied in the United States (U.S.) for the treatment of hepatic veno-occlusive disease (VOD), a rare, potentially life-threatening, early complication in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy. The three presentations include an update from an ongoing treatment investigational new drug (T-IND) study in the U.S., as well as updates from a number needed to treat (NNT, an epidemiological measure of effectiveness) analysis from a historically controlled pivotal Phase 3 trial in patients undergoing HSCT therapy, and from an international defibrotide compassionate use program.

Data from the three defibrotide studies will be presented today in an oral abstract session at the 2015 BMT (Bone Marrow Transplantation) Tandem meetings, the combined annual meetings of the American Society of Blood and Marrow Transplantation (ASBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR), in San Diego, California. BMT Tandem is one of the largest international forums dedicated specifically to HSCT.

"VOD is a potentially life-threatening complication in patients undergoing HSCT therapy, and there are currently no approved therapies for VOD in the U.S," said Jeffrey Tobias, M.D., executive vice president and chief medical officer of Jazz Pharmaceuticals. "The data presented at the BMT Tandem meetings build upon existing evidence showing that, when recognized and diagnosed, severe VOD may be effectively treated with defibrotide. The data also provide additional information on defibrotide's efficacy and safety profile in important subgroups of patients such as children, adults, and allograft and autograft recipients."

In 2014, Jazz Pharmaceuticals acquired the rights to defibrotide in the U.S. and other markets in North America, South America and Central America. Defibrotide has a Fast Track regulatory path designation in the U.S. for the treatment of severe VOD. The company has initiated a rolling new drug application submission to the U.S. Food and Drug Administration for defibrotide for the treatment of severe VOD and anticipates completing the submission in the first half of 2015.

In Europe, defibrotide is marketed under the name Defitelio®▼ (defibrotide). Defitelio is the first and only licensed product in Europe for the treatment of severe VOD in patients over one month of age undergoing HSCT therapy.

The three defibrotide oral presentations will be presented today during the BMT Tandem "Scientific" meeting, Session F - Allogeneic Transplants & Supportive Care & Graft Processing in the Harbor Ballroom ABC at the Manchester Grand Hyatt from 4:45 pm to 6:45 pm PST. Details are as follows:

• Paul G. Richardson, M.D., "Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease: An Analysis of Clinical Benefit As Determined By Number Needed to Treat (NNT) to Achieve Complete Response and to Improve Survival." Dr. Richardson and co-authors will present an NNT analysis from a Phase 3 study that formed the basis of the approval of defibrotide for the treatment of severe VOD following HSCT in the European Union. Dr. Richardson is the clinical program leader and director of clinical research at the LeBow Institute for Myeloma Therapeutics and the Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute and the RJ Corman Professor of Medicine at Harvard Medical School.
• Paul G. Richardson, M.D., "Updated Results from the Ongoing US Treatment IND Study Using Defibrotide for Patients with Hepatic Veno-Occlusive Disease." Researchers will present an interim analysis update from the T-IND study, which is gathering data on the safety and efficacy of defibrotide in patients with severe and non-severe VOD post-HSCT, as well as post-chemotherapy.
• Selim Corbachioglu, M.D., "Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease: An Update from the International Compassionate Use Program in 710 Patients." Dr. Corbachioglu, of the University of Regensburg, Germany, will present the final results of a large, international defibrotide compassionate use program conducted from 1998-2009 in Europe, the U.S., Asia and the Middle East.

SOURCE Jazz Pharmaceuticals plc