First Patient Enrolled in Jazz Pharmaceuticals plc (JAZZ) Phase 3 Trial of Defibrotide for the Potential Prevention of VOD

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) announced that the first patient has been enrolled in a Phase 3 clinical trial comparing the efficacy and safety of defibrotide versus best supportive care (BSC) in the prevention of hepatic veno-occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or at very high risk of developing VOD.  The defibrotide clinical trial will be conducted across approximately 100 medical centers in the United States (U.S.), Canada, Asia Pacific and countries in the European Union (EU).

“The initiation of this trial is another step forward in Jazz’s development program for defibrotide as a treatment option for patients who are at high risk or very high risk of developing this life-threatening condition,” said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer of Jazz Pharmaceuticals.  “If the data from this trial are positive, they may be used to pursue regulatory approval of a label expansion for defibrotide in both the U.S. and EU to include the prevention of hepatic VOD in high risk patients following HSCT.”

The Phase 3 trial is a randomized, open-label, multi-center trial with an adaptive design comparing the efficacy of defibrotide vs. BSC in the prevention of hepatic VOD.  The trial is expected to enroll approximately 400 adult and pediatric patients undergoing HSCT who are at high risk or very high risk of developing VOD. High (or very high) risk patients are identified due to the clinical regimen required to prepare them for HSCT, as well as their prior medical history and concomitant disease. The adaptive design allows for an interim analysis with pre-defined stopping points for early success or failure as well as the option to increase enrollment if needed to preserve the statistical power of the trial.

Shares of Jazz Pharmaceuticals closed today at $115.57, down $1.73 or -1.47%. JAZZ has a 1-year high of $160 and a 1-year low of $95.80. The stock’s 50-day moving average is $108.38 and its 200-day moving average is $122.64.

On the ratings front, JAZZ has been the subject of a number of recent research reports. In a report issued on January 12, Cantor analyst William Tanner reiterated a Buy rating on JAZZ, with a price target of $187, which represents a potential upside of 62% from where the stock is currently trading. Separately, on January 11, BMO’s Gary Nachman reiterated a Buy rating on the stock and has a price target of $192.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, William Tanner and Gary Nachman have a yearly average return of 3.8% and 8.7% respectively. Tanner has a success rate of 57% and is ranked #1459 out of 4349 analysts, while Nachman has a success rate of 56% and is ranked #897.

Overall, 6 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $173.00 which is 50% above where the stock opened today.

Jazz Pharmaceuticals Plc operates as a specialty biopharmaceutical company, which focuses on the identification, development and commercialization of pharmaceutical products. It has a diverse portfolio of products in the areas of narcolepsy, oncology, pain and psychiatry. Its product portfolio includes: XYREM, ERWINAZETM, PRIALT, intrathecal infusion, FAZACLO and LUVOX CR.


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