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FDA Approves Rare Liver Disorder Drug

March 30, 2016

The FDA has approved Defitelio (defibrotide sodium, Jazz Pharmaceuticals) to treat patients who develop hepatic veno-occlusive disease (VOD) with additional liver or kidney abnormalities after hematopoietic stem cell transplantation (HSCT). This is the first hepatic VOD treatment to be approved by the FDA.

VOD is a rare and life-threatening liver condition. Fewer than 2% of patients develop severe hepatic VOD after HSCT, but up to 80% of patients that do develop the condition do not survive.

The efficacy of defibrotide sodium was investigated in two prospective clinical trials and one expanded access study. Patients included in the three studies (N = 528) had been diagnosed with hepatic VOD with liver or kidney abnormalities after HSCT. In patients treated with defibrotide sodium, overall survival 100 days after HSCT was 38% to 45%, compared with an expected overall survival rate of 21% to 31% in patients receiving only supportive care or treatments other than defibrotide sodium.

Common side effects include hypotension, diarrhea, vomiting, nausea, and nosebleeds. Serious potential side effects of treatment include bleeding (hemorrhage) and allergic reactions. Defibrotide sodium should not be used in patients who are having bleeding complications or who are taking blood thinners or other medicines that reduce the body’s ability to form clots.

Reference: FDA approves first treatment for rare disease in patients who receive stem cell transplant from blood or bone marrow [press release]. March 30, 2016.

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