Jazz Pharmaceuticals announced that researchers will present the results of a number needed to treat (NNT; an epidemiological measure of effectiveness) analysis from a historically controlled phase 3 clinical trial evaluating the use of defibrotide for the treatment of severe hepatic veno-occlusive disease (severe VOD or sVOD) in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy.

Researchers will also present updates from an international compassionate use programme and an expanded access / treatment investigational new drug (IND) study in the United States (US).  These two updates will include data evaluating the use of defibrotide in the treatment of VOD, a rare, early complication in patients undergoing HSCT.  These data will be presented in posters at the American Society of Hematology (ASH) 56th Annual Meeting and Exposition in San Francisco, California, taking place from December 6 - 9, 2014.

Earlier this year, Jazz Pharmaceuticals acquired the rights to defibrotide in the US and other markets in North America, South America and Central America.  Defibrotide has a fast track regulatory path designation in the US.  The company plans to initiate a rolling new drug application (NDA) submission to the US Food and Drug Administration for defibrotide for the treatment of severe VOD by the end of the year 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 licenced product in Europe for the treatment of severe VOD in patients over one month of age undergoing HSCT therapy.

"The analyses being presented at ASH this year will further enhance our understanding of defibrotide's activity in the treatment of VOD overall, and of severe VOD in particular," said Paul G. Richardson, MD, clinical programme 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.  "Importantly, the NNT analysis to be presented will provide additional information on the clinical impact of treatment, further supporting the use of defibrotide as a potential therapeutic option for patients in the treatment of severe VOD."

In addition, results from an analysis supported by Jazz Pharmaceuticals that modeled the impact of using paediatric-inspired treatment protocols compared with hyper-CVAD protocols on outcomes for adolescents and young adults with Philadelphia negative acute lymphoblastic leukaemia (ALL) will be presented in a poster session on December 6, 2014.

"We are pleased to be able to contribute to the scientific presentations at this year's ASH meeting and to provide physicians with additional information regarding the treatment of patients with severe VOD and ALL," said Jeffrey Tobias, MD, executive vice president and chief medical officer of Jazz Pharmaceuticals.

Defibrotide is marketed under the name Defitelio(defibrotide) in Europe. In October 2013, the European Commission granted marketing authorisation under exceptional circumstances for Defitelio for the treatment of severe hepatic VOD in patients undergoing HSCT therapy.  It is indicated in patients over one month of age.  Defitelio is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.