6. Defitelio

Active Ingredient: defibrotide
Disease: severe hepatic veno-occlusive disease
Peak sales estimate: $350 million to $480 million
Approved: March 30
Company: Jazz Pharmaceuticals

The scoop: Upon its March approval, Jazz Pharma’s Defitelio became the first drug marketed in the U.S. to treat severe hepatic veno-occlusive disease, providing a significant therapeutic advance to a small group of patients. Jazz bought European rights to the drug in 2013 with its $1 billion purchase of Gentium and U.S. rights the next year with a deal worth up to $250 million with Italy’s Sigma-Tau. Hepatic VOD, a condition in which some of the veins in the liver become blocked, develops following stem cell transplants and is often fatal. At the time of the approval, FDA’s Richard Pazdur said the med “fills a significant need” among those who undergo such transplants. Approved in Europe back in 2013, Defitelio brought in $70 million in 2015 sales for Jazz; the company expects that figure to be $105 million to $120 million this year. The FDA first rejected Defitelio in 2011, raising some doubts about its data that Jazz ultimately answered with the regulatory nod. -- Eric Sagonowsky

6. Defitelio

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