Italy-based Gentium and Link Healthcare announced that defibrotide has been designated as an orphan drug for the treatment of hepatic veno-occlusive disease (VOD).
The sponsor of Defibrotide is Link Healthcare, Gentium's exclusive distribution partner 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); Gentium will now work with Link to commence the Marketing Application process in Australia.
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 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.