Jazz Pharmaceuticals announced that it has started the rolling submission of a New Drug Application (NDA) for defibrotide as treatment for severe hepatic veno-occlusive disease (VOD) in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy.
Defibrotide is approved and indicated in the EU as treatment for severe hepatic VOD in patients one month old and above undergoing HSCT therapy. The drug has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of VOD. The company announced earlier this year that it will acquire rights to defibrotide from Sigma-Tau Pharmaceuticals through Jazz’s subsidiary Gentium.
Veno-occlusive disease is an early complication in patients under HSCT therapy. The therapy is performed to treat hematological malignancies, certain tumors, and other non-malignant disorders. Severe VOD can be deadly and is linked with multi-organ failure. The condition is fatal in 80 percent of patients.
Jazz presented analysis of positive results from a Phase 3 trial of defibrotide in severe hepatic VOD at the recent American Society of Hematology (ASH) 56th Annual Meeting and Exposition held in San Francisco, California.
Jeffrey Tobias, EVP and CMO of Jazz Pharmaceuticals, said, “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. …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 U.S.”
The FDA’s Fast Track designation enables faster development and review of drugs that treat serious, deadly conditions and that address significant unmet medical needs. The Fast Track ‘rolling submission’ process permits a company to submit parts of its New Drug Application (NDA) for review as soon as they are completed instead of waiting until all sections of the application are available before submitting them as a whole.