The U.S. FDA approved defibrotide sodium for adults and children with hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after they have received hematopoietic cell transplantation (HCT). This is the first FDA-approved treatment for severe hepatic VOD. Hepatic VOD is a condition in which some of the veins in the liver become blocked, causing swelling and a decrease in blood flow inside the liver, which may lead to liver damage.
The approval was based on the results of three studies – two prospective clinical trials and one expanded access study – that examined overall survival (OS) in 528 patients who had undergone HCT. In the three studies, 38 to 45 percent of patients treated with defibrotide sodium were alive at 100 days post-HCT. Based on published reports and analyses of patient-level data, the expected survival rates 100 days after HCT would be 21 to 31 percent for patients with severe hepatic VOD who received only supportive care or interventions other than defibrotide, the study researchers noted.
The most common AEs associated with defibrotide sodium include hypotension, diarrhea, vomiting, nausea, and epistaxis. Serious AEs include hemorrhage and allergic reactions.
Patients with bleeding complications or those taking blood thinners or other medications to reduce blood clots should not receive defibrotide sodium.
Source: U.S. FDA press release, March 30, 2016.