VILLA GUARDIA (COMO), Italy, Aug 17, 2011 (GlobeNewswire via COMTEX) -- Gentium S.p.A. /quotes/zigman/97116 GENT -2.01% (the "Company") today announced that it has voluntarily withdrawn the New Drug Application (NDA) for Defibrotide following recent correspondence from the United States Food and Drug Administration (FDA) identifying numerous "Refuse to File" issues regarding the Company's NDA submission.
In their initial review, the FDA raised concerns regarding the completeness of the datasets for both the treatment and prevention studies. The FDA requested that the Company conduct additional quality reviews of the original datasets and databases. The FDA also requested additional details regarding the conduct and monitoring of the trials by the independent review committee.
"Given the number of issues raised by the FDA and the short deadline we have to address such issues, we felt that it was appropriate to voluntarily withdraw our NDA submission for Defibrotide," stated Dr. Khalid Islam, Chairman and Chief Executive Officer of Gentium S.p.A. Dr. Islam added, "It is our plan to address these concerns as quickly as possible and to work closely with the FDA to provide an updated and complete NDA."
Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as a significant complication of stem cell transplantation. Certain high-dose conditioning regimens used as part of stem cell transplant (SCT) can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of VOD in the US or the EU.
Gentium S.p.A., located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of drugs to treat and prevent a variety of diseases and conditions, including vascular diseases related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status by the U.S. FDA and Orphan Medicinal Product Designation by the European Commission both to treat and to prevent VOD and Fast Track Designation by the U.S. FDA to treat VOD.