Jazz eyes a quick FDA nod for its $1B rare disease drug

Jazz Chief Medical Officer Dr. Karen Smith

Jazz Pharmaceuticals ($JAZZ) secured the FDA's priority review for a long-delayed rare disease treatment, accelerating the path of a drug acquired in a $1 billion buyout.

The drug, defibrotide, is designed to relieve symptoms of a vein-blocking disorder called hepatic veno-occlusive disease (VOD), which sometimes occurs after stem cell transplants. VOD is a rare and early complication in the stem cell process, Jazz said, with a mortality rate exceeding 80%.

The FDA's priority program shortens the standard 10-month review down to 6 months, putting defibrotide in line for a final decision by March 31. The drug won European approval in 2013 and is marketed overseas as Defitelio.

Jazz picked up the European rights defibrotide in its 2013 buyout of Gentium, paying $1 billion in cash with hopes of expanding the drug's potential. The following year, Jazz paid $75 million up front and promised up to $175 million more to Sigma-Tau in exchange for the U.S. rights, betting it could reverse defibrotide's record of regulatory trouble.

The FDA rejected Gentium's first pitch for approval in 2011, raising concerns about the completeness and quality of its data and asking for more details about how supporting trials were conducted and monitored. Jazz believes it has now satisfied those requests, hoping to win approval "as quickly as possible, as there are no other approved therapies for treating this rare, often fatal complication" of hematopoietic stem-cell transplantation, Chief Medical Officer Dr. Karen Smith said in a statement.

Beyond VOD, defibrotide has potential in graft-versus-host disease, Jazz has said, an indication for which the treatment has orphan drug designation in the EU.

- read the statement

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