Gentium’s exclusive distribution partner in Australia, Link Healthcare has received orphan drug designation for Defibrotide for treatment of Hepatic Veno-Occlusive Disease (VOD). Gentium will now work with Link to commence the Marketing Application process in Australia.

The main characteristics of the orphan drug policy in Australia are: it is intended for drugs which aim to treat diseases with a prevalence of 2,000 patients/subjects or less in the Australian population (23 million); it allows for a waiver of application and evaluation and no annual registration fees; it provides five-year period of exclusivity (under consideration by the Australian jurisdiction).

Veno-occlusive disease (VOD) 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 transplantation can damage the lining cells of hepatic blood vessels and result in VOD, a blockage of the small veins in 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). Stem cell transplantation is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children.

Gentium S.p.A. 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.