Jazz begins rolling NDA submission for defibrotide to treat severe hepatic VOD

PBR Staff Writer Published 15 December 2014

Ireland-based Jazz Pharmaceuticals has started rolling submission of a new drug application (NDA) to the US Food and Drug Administration (FDA) for defibrotide to treat severe hepatic veno-occlusive disease (VOD) in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy.

The US FDA has also granted fast track designation to defibrotide for the treatment of severe VOD.

In its severe form, VOD can be life-threatening and is related with multi-organ failure, while it is fatal in more than 80% of patients

Jazz Pharmaceuticals executive vice-president and chief medical officer Jeffrey Tobias said: "Our start of the NDA submission for defibrotide marks an important step forward in our efforts to provide a treatment option for patients in the US who develop this rare, life-threatening complication of HSCT.

"We expect to complete the submission of the NDA in the first half of 2015, at which time we will be requesting a Priority Review of the application, and we will continue to work closely with the FDA as we seek approval of the NDA.

"As part of our commitment to ensure that eligible patients have access to defibrotide as we pursue US approval, we will continue to provide patients access to defibrotide through an expanded access treatment protocol that is open under an ongoing investigational new drug application in the US."

Earlier in 2014, the company acquired the rights to defibrotide in the US and other markets in North America, South America and Central America.

The company markets defibrotide in Europe under the name Defitelio (defibrotide), which is the first and only licensed product in Europe to treat severe hepatic VOD in patients over one month of age undergoing HSCT therapy.

According to the company, defitelio is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

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Jazz begins rolling NDA submission for defibrotide to treat severe hepatic VOD