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Jazz Pharma receives FDA approval for rare veno occlusive disease

Posted on over 1 year ago by Gerry Kennedy

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Jazz Pharmaceuticals (NASDAQ: JAZZ) has announced that the US Food and Drug Administration (FDA) has granted marketing approval for Defitelio (defibrotide sodium) for the treatment of adult and paediatric patients with hepatic veno-occlusive disease.

Also called sinusoidal obstruction syndrome (SOS), Defitelio is indicated for the treatment of this rare, and often fatal, liver disease in patients with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT). This transplantation is a procedure performed in patients to treat certain blood or bone marrow cancers.

It is the first FDA-approved therapy for the treatment of hepatic VOD. The treatment was approved in the EU Shipments of Defitelio to distribution channels are expected to commence within a week.

This approval follows the publication of efficacy data from three clinical studies in patients with the disease who were treated with Defitelio. For example, the results showed that between 38% and 45% of patients receiving Defitelio were still alive 100 days after transplantation, compared to the norm of between 21% and 31% of patients. Its safety was also emphasised with the incidence of adverse events relatively low.

Bruce Cozadd, chairman and chief executive officer of Jazz pharmaceuticals, comments: “FDA’s approval of Defitelio underscores the importance of [the drug] to children and adults and the first and only proven treatment for this rare and often deadly complication of stem-cell transplantation. 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.”

Paul G Richardson, academic and director of clinical research at the LeBow Institute for Myeloma Therapeutics, says: “VOD/SOS is a devastating condition, which can develop without warning after stem-cell transplantation and can progress rapidly causing severe kidney or lung dysfunction and lead to multi-organ failure… Importantly, Defitelio provides transplant teams with the first approved treatment option that can help return patients to the road to recovery.”

Richard Padzur, director of the office of haematology and oncology products at the FDA, indicates: “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.”

Sean Murray