News Feature | July 3, 2014

Jazz To Acquire Rights To Sigma-Tau's Defibrotide

By Estel Grace Masangkay

Jazz Pharmaceuticals announced that it has signed into a definitive agreement to acquire defibrotide from Sigma-Tau Pharmaceuticals. Jazz will accomplish the transaction through its subsidiary Gentium, which it acquired earlier this year, and the rights will be applicable in the Americas and in the U.S.

Defibrotide, marketed in the EU as Defitelio, is a drug indicated for the treatment of severe hepatic veno-occlusive disease (VOD) in patients more than 1 month old and undergoing hematopoietic stem cell transplantation (HSCT) therapy. The drug has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and the Korean Ministry of Food and Drug Safety (MFDS) among others. The company is currently conversing with the FDA, which has also granted Fast Track designation for defibrotide, about the submission of a New Drug Application (NDA) for defibrotide as severe VOD treatment.

Sigma-Tau currently holds commercial rights to defibrotide in the Americas under an agreement with Gentium. As part of the new agreement, Jazz will make an upfront payment of $75 million to Sigma-Tau at the close of the transaction. Sigma-Tau would also be eligible for milestone payments of up to $25 million upon the FDA acceptance of the drug’s first NDA. The company is also eligible to receive an additional $150 million depending on the timing of potential FDA approval.

Hepatic veno-occlusive disease is an early complication in patients undertaking hematopoietic stem cell transplantation (HSCT) therapy. VOD in its severe form can be deadly and is linked with multi organ failure. The disease is fatal in more than 80 percent of patients. Five to 15 percent of patients undergoing HSCT will develop VOD.

Russell Cox, EVP and COO of Jazz Pharmaceuticals, said, “We look forward to ongoing discussions with the FDA as we continue our efforts toward submission of an NDA for defibrotide in the U.S. Patients in the U.S. with severe VOD have a critical unmet medical need, and we believe that defibrotide has the potential to become an important treatment option for these patients.”

The companies expect the transaction to close in the third quarter of 2014.