FDA OKs First Rx for Rare Liver Condition

Defitelio (defibrotide sodium), from Jazz Pharmaceuticals, approved to treat severe liver condition

/ Author:  / Reviewed by: Anyssa Garza, PharmD

(RxWiki News) The US Food and Drug Administration (FDA) has approved Defitelio (defibrotide sodium) to treat adults and children with severe hepatic (liver) veno-occlusive disease (VOD).

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

This approval will allow Defitelio to treat patients who may develop liver VOD after receiving chemotherapy and hematopoietic stem cell transplantation (HSCT). HSCT is a procedure performed in some patients to treat certain blood or bone marrow cancers.

Liver VOD occurs when some of the veins in the liver become blocked. The reduced blood flow to the liver may lead to liver damage. If liver VOD is severe, the lungs and kidneys may be damaged as well.

Trial data suggested that Defitelio increased the number of patients who were alive 100 days after HSCT (overall survival), compared to other treatments.

The most common side effects of Defitelio included abnormally low blood pressure, diarrhea, vomiting, nausea and nosebleeds.

A potential serious side effect of Defitelio is bleeding. Defitelio should not be used in patients who are having bleeding complications or who are taking blood thinners or medications that reduce the ability to form clots.

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Review Date: 
March 31, 2016
Last Updated:
April 7, 2016