Italy's Gentium has suffered a major setback after it emerged that the European Medicines Agency is likely to turn down defibrotide, its investigational treatment for hepatic veno-occlusive disease.
The company noted that following an oral explanation to the EMA, and based on preliminary feedback from the agency's Committee for Medicinal Products for Human Use, Gentium expects an opinion recommending against approval of defibrotide to treat and prevent hepatic VOD in patients undergoing hematopoietic stem cell transplantation therapy. Gentium added that while not a final decision, it "considers it unlikely that this position will change before the formal vote is undertaken next month".
The Como-based firm says it may appeal a negative opinion and chief executive Khalid Islam said "we await the official decision and the formal report, which should provide us specifics on any additional requirements leading to the approval of defibrotide in Europe". He added that "we remain confident 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 of VOD, an unmet medical need where no known agents are currently approved".
In August 2011, Gentium pulled its US marketing application for defibrotide after it became clear that the Food and Drug Administration would not accept the filing.