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."