$GENT Announces NDA Submission for Defibrotide

VILLA GUARDIA (COMO), Italy, Jul 6, 2011 (GlobeNewswire via COMTEX) -- Gentium S.p.A. GENT -0.10% (the "Company") today announced that it has submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Defibrotide for the treatment of hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem-cell transplantation (HSCT).

"If approved, Defibrotide would become the first drug approved for the treatment of VOD, a serious and potentially fatal complication of hematopoietic stem-cell transplantation," stated Dr. Khalid Islam, Chairman and Chief Executive Officer of Gentium S.p.A. "Defibrotide has been used as an investigational drug in more than 250 clinics across 33 countries in the on-going global named-patient programs, and the United States expanded access study. We are committed to working with the FDA on the approval of Defibrotide and with our partner, Sigma-Tau Pharmaceuticals, Inc., on the future commercialization of Defibrotide."

Gregg Lappointe, CEO of Sigma-Tau Pharmaceuticals, Inc., commented, "We congratulate Gentium on reaching this important milestone and moving toward U.S. regulatory approval for Defibrotide. We are committed to working closely with Gentium to ensure a successful launch in the U.S. for this potentially lifesaving therapeutic candidate."

The efficacy of Defibrotide to treat hepatic VOD in HSCT patients is supported by data from a multi-center Phase 3 historically-controlled trial, evaluating Defibrotide for the treatment of severe VOD (patients with VOD and multi-organ failure), a Phase 2 dose finding study, and interim data reported from the ongoing Phase 3 expanded access U.S. Treatment IND program in patients with severe hepatic VOD. Additional data include a Phase 3 randomized controlled study of Defibrotide in the prevention of hepatic VOD in pediatric HSCT patients. Defibrotide has generally been well-tolerated in the clinical setting, and results in more than 1,300 patients to date have shown that generally Defibrotide does not appear to increase the risk of complications in HSCT patients.

Defibrotide has been granted orphan drug designation by the FDA to treat severe VOD. Orphan drug designation is a special status given to products for diseases or conditions that affect fewer than 200,000 people in the United States. If the FDA approves the NDA for this use of Defibrotide, before approving a NDA filed by anyone else for the same use, the orphan drug status will grant us limited market exclusivity for seven years from the date of the FDA's approval of our NDA.

Defibrotide has also been granted "fast track" designation by the FDA for the treatment of severe VOD in recipients of stem cell transplants. Fast track is a process designed to facilitate the development and expedite the review of drugs that are intended to treat serious diseases and fill an unmet medical need.

Gentium recently filed a marketing authorization application with the European Medicines Agency (EMA) for the prevention and treatment of hepatic veno-occlusive disease (VOD) in adults and children undergoing stem-cell transplantation therapy. The application was accepted and granted an accelerated assessment by the EMA on May 25, 2011