Defibrotide sodium has been approved to treat hepatic veno-occlusive disease (VOD) in patients with renal or pulmonary dysfunction following a hematopoietic stem cell transplantation, the Food and Drug Administration has announced.
The drug, which will be marketed as Defitelio by Jazz Pharmaceuticals, was tested in two prospective clinical trials and an expanded access study that included a total of 528 patients with hepatic VOD and multiorgan dysfunction following a transplantation. All patients received 6.25 mg/kg doses of the drug intravenously, every 6 hours until resolution of VOD. The percentages of patients surviving more than 100 days after receiving a stem cell transplantation in each of the studies were 38%, 44%, and 45%, respectively, according to a statement from the FDA.
Contraindications for taking the drug are concurrent use of anticoagulants or fibrinolytic therapies.
Hypotension, diarrhea, vomiting, nausea, and epistaxis are the most common adverse reactions to the drug.
Full prescribing information is available at the FDA website.