- Ireland-based Jazz Pharmaceuticals on Wednesday won FDA approval of its rare liver disease drug defibrotide, making it the first approved treatment for heaptic veno-occulsive disease (VOD) in the U.S.
- The drug had previously been granted priority review and orphan drug status. VOD is a complication of hemotopoietic stem-cell transplantation and can lead to permanent liver damage. As many as 8 out of 10 patients who develop the most severe form of hepatic VOD die, according to the FDA.
- The drug already won approval in Europe and is marketed as Difetelio. Jazz originally acquired the drug through the accusation of Gentium SpA, Reuters reports.
Each year, approximately 20,000 people in the US undergo stem-cell transplantation, with very high levels of therapeutic success. However, up to 14% of patients develop VOD, which can lead to multiple organ failures and possible death.
Jazz submitted data from three studies, including one phase 3 study, highlighting defibrotide's efficacy and safety. In the expanded access study, which included 351 patients with VOD, the survival rate after day 100 was 45%.
"Defitelio is a clinically significant therapeutic advance because it is a potentially curative intervention for patients with VOD, which may save lives with a single course of therapy. Before today, patients in the U.S. had no approved options," said Jazz CEO Bruce Cozadd.
The company will begin shipping the drug to its distribution network within a week.