Positive EU orphan opinion for Gentium’s defibrotide

Italian biotech company Gentium (Nasdaq: GENT) says that the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the company's application for orphan medicinal product status for defibrotide for the prevention of Graft versus Host Disease (GvHD).

The positive opinion of the COMP has now been forwarded to the EU commission for final approval and publication in the EU community register. The drug is currently under review at the EMA to treat and prevent hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy (The Pharma Letter February 22).

Khalid Islam, Gentium's chairman and chief executive, said: "Graft versus Host Disease is one of the most important and potentially fatal complications of hematopoietic stem cell transplantation (HSCT) that afflicts up to 50% of all donor transplant patients. We are pleased to have received a positive opinion for this second indication from the COMP for orphan drug status in Europe. Based on its differentiated and novel mechanism of action when compared with currently used drugs and the encouraging clinical results obtained in prevention of GvHD (Corbacioglu et al, Lancet 2012) we believe that Defibrotide will address a significant unmet medical need for this potentially life-threatening disease. We look forward to planning and initiating additional studies to explore the full potential of defibrotide in this indication."