The US Food and Drug Administration (FDA) has granted marketing approval for Jazz Pharmaceuticals‘ Defitelio (defibrotide sodium) to treat hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

VOD is a rare complication of HSCT, which is often characterised by sudden weight gain, hepatomegaly (abnormally enlarged liver), and elevated bilirubin.

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It is a devastating condition, which is believed to develop without warning after stem-cell transplantation and can progress rapidly, causing severe kidney or lung dysfunction and can lead to multi-organ failure.

HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous hematologic disorders such as leukaemia, lymphoma and aplastic anaemia, as well as congenital immunodeficiency and autoimmune disorders.

The biopharmaceutical company said that 80mg/ml Defitelio injection is indicated for the treatment of adult and paediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome (SOS).

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"Defitelio is a clinically significant therapeutic advance because it is a potentially curative intervention for patients with VOD, which may save lives with a single course of therapy."

Jazz Pharmaceuticals CEO Bruce Cozadd said: "FDA’s approval of Defitelio underscores the importance of Defitelio to children and adults as the first and only proven treatment for this rare and often deadly complication of stem-cell transplantation.

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"Defitelio is a clinically significant therapeutic advance because it is a potentially curative intervention for patients with VOD, which may save lives with a single course of therapy.

"Before today, patients in the US had no approved options."

Approval is based on data from three clinical studies, which evaluated the efficacy of Defitelio in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT.

Defitelio also received orphan drug designation for the treatment of hepatic VOD.