Veno Occlusive Disease or VOD, a condition in which some of the veins in the liver are blocked, is a complication of cancer treatment given prior to stem cell transplantation. Severe VOD is associated with multiple-organ failure and high rates of morbidity and mortality. Currently, there are no approved treatments for VOD by either the U.S. FDA or the European Medicines Agency. One of the promising experimental drugs for the treatment of veno-occlusive disease is Defibrotide, developed by Italian drugmaker Gentium S.p.A. (GENT: Quote).
For readers who are new to Gentium, here's a brief overview of the company's pipeline and the upcoming events to watch out for...
Defibrotide is the company's most advanced product candidate, which has completed a phase III trial for treatment of severe VOD in the U.S., Canada and Israel and a phase II/III pediatric trial in Europe for the prevention of VOD.
Gentium submitted a New Drug Application to the FDA for Defibrotide for the treatment of hepatic veno-occlusive disease in adults and children undergoing hematopoietic stem cell transplantation in July of 2011. However, with the FDA raising concerns regarding the completeness of the datasets for both the treatment and prevention studies, the company voluntarily withdrew the Defibrotide NDA last August. Since then, Gentium has engaged contract research organizations, or CROs, and several outside consultants to assist in addressing the issues raised by the FDA, and is working towards a resubmission of Defibrotide NDA.
An application seeking marketing approval for Defibrotide to treat and prevent hepatic veno-occlusive disease in adults and children undergoing haematopoietic stem cell transplantation therapy was submitted to the EMA by Gentium in May of 2011. The EMA's Committee for Medicinal Products for Human Use, or CHMP, which reviewed the Defibrotide application, issued the LoQs (List of Questions) last September, seeking some clarifications from the company regarding the drug.
In February of this year, Gentium responded to the EMA's List of Questions for Defibrotide application. However, as further clarification was required, the CHMP issued a LoOI, or List of Outstanding Issues, for the Defibrotide application in May of 2012.
In line with the regulatory timetable, last week (June 21), the company submitted its response to the List of Outstanding Issues received from the CHMP. If no further explanation or clarification is required, a recommendation regarding the approval of Defibrotide could be issued by the CHMP as early as the third quarter of 2012, according to Gentium.
Even though Defibrotide is not approved anywhere in the world, it continues to be distributed through the company's cost recovery and named-patient programs. The volume of Defibrotide distributed has been on the rise over the years, thanks to increased awareness of Defibrotide and the distribution partnerships established by Gentium.
Defibrotide has been given "orphan" status by the FDA and the EMA, as well as fast-track product" designation by the FDA.
The right to market Defibrotide in the United States, upon FDA approval, to treat and prevent VOD is licensed to Sigma-Tau Pharmaceuticals Inc. In certain European countries and in certain territories in other parts of the world, on a named-patient supply basis, IDIS Ltd. is the exclusive supplier of Defibrotide.
Gentium also has license and/or supply and distribution agreements with specialized regional partners for the distribution of Defibrotide on a named-patient supply basis in Turkey, Israel, Palestinian Authority, Australia, New Zealand, Sweden, Denmark, Norway, Iceland, Finland, Latvia, Lithuania and Estonia.
As recently as May 2, 2012, Gentium appointed PharmaSwiss S.A., a division of Valeant Pharmaceuticals International Inc. (VRX) as the exclusive distributor of Defibrotide in Albania, Bosnia, Bulgaria, Croatia, Cyprus, Czech Republic, Greece, Hungary, Macedonia, Montenegro, Poland, Romania, Serbia, Slovenia and Slovakia.
Apart from the revenue generated through distribution of Defibrotide, Gentium also recognizes revenue from the sale of APIs, or Active Pharmaceutical Ingredients, including urokinase and sulglicotide, produced at the company's manufacturing plant in Italy. Urokinase, an API made from human urine, is used to dissolve fibrin clots while sulglicotide, developed from swine duodenum, is used for ulcer healing and gastrointestinal protection.
A quick look at the company's balance sheet...
In the first quarter ended March 31, 2012 Gentium incurred a net loss of EUR 0.91 million or EUR 0.06 per share on revenues of EUR 5.66 million. That compared with a net income of EUR 1.70 million or EUR 0.114 per share on revenues of EUR 6.05 million. Defibrotide net sales through the named-patient and cost recovery programs in the recent first quarter rose 28% to EUR 4.93 million.
The company ended the first quarter of 2012 with EUR 10.68 million in cash.
Gentium was initially listed on the Amex under ticker 'GNT', following its IPO in June 2005 at $9.00 per ADS. In May 2006, the listing was switched to the NASDAQ National Market System under a new ticker 'GENT'.
GENT has thus far hit a 52-week low of $5.31 and a 52-week high of $10.25. The stock closed Friday's trading at $9.26, down 1.49%.
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by RTT Staff Writer
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