Gentium announced that it has been informed by the European Medicines Agency's, or EMA, Committee for Medicinal Products for Human Use, or CHMP, that it has adopted a negative opinion, recommending the refusal of the marketing authorization for Defibrotide to treat and prevent hepatic veno-occlusive disease, or VOD, in adults and children undergoing hematopoietic stem cell transplantation therapy. Gentium remains convinced of the favourable benefit/risk profile of Defibrotide, which is the only option recommended by the European Group for Blood and Marrow Transplantation for the treatment VOD, an unmet medical need where no agents are currently approved. In accordance with European regulations, the company plans to appeal the EMA's decision and request a re-examination of the CHMP opinion.
- Disease & Medical Conditions
- European Medicines Agency