VILLA GUARDIA, Italy, April 16, 2013 (GLOBE NEWSWIRE) -- Gentium S.p.A. (Nasdaq:GENT) (the "Company") today announced that it has requested a re-examination of the negative opinion adopted, by the European Medicines Agency's ("EMA") Committee for Medicinal Products for Human Use ("CHMP"), on March 22nd, regarding the Marketing Authorisation (MAA) submitted for Defibrotide to treat and prevent hepatic veno-occlusive disease ("VOD") in adults and children undergoing hematopoietic stem cell transplantation therapy.
In accordance with European regulations, the Company has 60 days to submit grounds for appeal and the CHMP has further 60 days to consider a revision of the initial opinion. The appeal involves a re-examination of the original MAA and must be based on the data already submitted.
Notwithstanding the CHMP's negative opinion, 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.
EU patients will continue to have access to Defibrotide, under a named patient program ("NPP") where available.
The European Group for Blood and Marrow Transplantation (EBMT) have also commented on the CHMP's recommendation not to grant a marketing authorization; www.ebmt.org.
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.
Gentium S.p.A., located in Como, Italy, is a biopharmaceutical company focused on the development and manufacture of drugs to treat and prevent a variety of diseases and conditions, including vascular diseases related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product Designation by the European Medicines Agency, both to treat and to prevent VOD, as well as Fast Track Designation by the U.S. FDA to treat VOD.
Cautionary Note Regarding Forward-Looking Statements
This release contains "forward-looking statements" that involve a number of risks and uncertainties the outcome of which could materially and/or adversely affect actual future results and the market price of Gentium's securities. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "potential" or "continue," the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results, including with respect to the possibility of any future regulatory approval, may differ materially from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20-F filed with the Securities and Exchange Commission under the caption "Risk Factors."