FDA approves Jazz Pharmaceuticals’ Defitelio

SILVER SPRING, Md. — The Food and Drug Administration has approved Jazz Pharmaceuticals’ Defitelio (defibrotide sodium), a first-in-class treatment for a rare, life-threatening liver condition in patients who’ve received a stem cell transplant from blood or bone marrow.  

 

Defitelio is indicated to treat hepatic veno-occlusive disease (VOD in patients with additional kidney or lung abnormalities that develops after a hematopoietic stem cell transplant (HCST). The condition causes some veins in the liver to be blocked, reducing blood flow in the liver. The most severe form of hepatic VOD can cause kidney and lung failure. Though only about 2% of HCST patients develop VOD, the mortality rate among those who do is 80%. 

 

“The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT,” said Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.  

 

The FDA granted priority review status to Defitelio and it received an orphan drug designation. The injectable drug’s distribution is expected to being within a week.