30 March 2016 - The U.S. FDA today approved Defitelio (defibrotide sodium) to treat adults and children who develop hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called hematopoietic stem cell transplantation (HSCT).

This is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition.

The FDA granted the Defitelio application priority review status, which facilitates and expedites the development and review of certain drugs in light of their potential to benefit patients with serious or life-threatening conditions. Defitelio also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for exclusivity to assist and encourage the development of drugs for rare diseases.

For more details, go to: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm493225.htm