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Jazz Pharmaceuticals Wins FDA Approval For Rare Liver Disease Drug

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Stem Cell Transplant

Defitelio is approved to treat patients with hepatic veno-occlusive disease (VOD), which can occur after a patient receives a hematopoietic stem cell transplantation.

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April 1, 2016 | by Sarah Massey, M.Sc.

At long last, Jazz Pharmaceuticals has received US Food and Drug Administration (FDA) approval for its rare liver disease drug, defibrotide. The drug – sold under the name Defitelio – was first rejected by the regulatory agency five years ago, amid concerns regarding data quality.

Defitelio is approved to treat patients with hepatic veno-occlusive disease (VOD), which can occur after a patient receives a hematopoietic stem cell transplantation. VOD is a rare but potentially life-threatening disease, and Defitelio is the first FDA-approved treatment for the condition.

It’s estimated that 20,000 blood or bone marrow transplants are performed in the US each year, with 10 percent of those patients developing severe VOD. Analysts expect the drug will be launched right away.

“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,” said Bruce Cozadd, CEO of Jazz Pharmaceuticals. “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 U.S. had no approved options.”



Jazz Pharmaceuticals acquired the European rights to defibrotide after its 2013 buyout of Gentium. The Ireland-based pharmaceutical company paid $1 billion in cash for the drug in the hopes that the drug would show potential.

In 2014, Jazz Pharmaceuticals paid $250 million for the US rights to drug, which were previously held by Sigma-Tau. Considering the FDA turned down Gentium – citing concerns about clinical trial data – when they applied for US approval for defibrotide in 2011, Jazz’s move to acquire the drug was a risky one.

This recent FDA approval was a big win for Jazz, as the drug has the potential to be very lucrative for the biotech. Defibrotide raked in $70.7 million in the European market in 2015, however the US price of the drug has yet to be announced.


Keywords: Liver Disease, Pharmaceutical Industry, Rare Disease


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