S.p.A., a biopharmaceutical company focused on the development and manufacture of drugs to treat and prevent a variety of diseases and conditions, has received the European Commission marketing authorization for Defitelio (defibrotide) for the treatment of severe hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy. Defitelio is the first approved treatment in the European Union for this life-threatening condition. This authorization is the formal endorsement of the positive opinion received from the European Medicine Agency’s (EMA) Committee for Human Medicinal Product (CHMP) on July 26, 2013. The company intends to begin commercialization of Defitelio in the European Union in December 2013.
“Today’s authorization of Defitelio represents an important milestone for Gentium,” said Dr Khalid Islam, chairman and chief executive officer of Gentium S.p.A. “As the first drug to be approved for the treatment of severe VOD, Defitelio will provide physicians with a life-saving option addressing a high unmet medical need for a disease with a high mortality rate, in excess of 80%. Gentium is proud of this achievement, which we believe will provide comfort and hope for patients and their families.”
“The decision allows Gentium to market Defitelio in the 28 Member States of the EU. We expect to begin commercial sales of Defitelio through our own sales force and through our distributor partners by the end of 2013,” said Adrian Haigh, SVP commercial operations and chief operating officer of Gentium GmbH.
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. At present there is no approved agent for the treatment or prevention of VOD in the United States or the European Union.