US regulators today approved the first treatment for a rare and life-threatening condition called hepatic veno-occlusive disease.

The US Food and Drug Administration has issued a green light for Defitelio (defibrotide sodium) to treat adults and children who develop hepatic VOD with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called haematopoietic stem cell transplantation (HSCT). 

Less than two percent of patients develop severe hepatic VOD after HSCT, but as many as 80 percent of patients who develop severe hepatic VOD do not survive. 

In clinical trials, 38 percent to 45 percent of patients treated with Defitelio were alive 100 days after HSCT. Evidence shows the expected survival rates 100 days after HSCT would be 21 to 31 percent for patients with severe hepatic VOD who received only supportive care or interventions other than Defitelio.  

“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 Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.  

The most common side effects of Defitelio include abnormally low blood pressure (hypotension), diarrhoea, vomiting, nausea and nosebleeds. Serious potential side effects that were identified include bleeding and allergic reactions.  

The drug was launched in Europe in 2014.