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Gentium shares wrecked by fresh round of regulatory woes for lead drug

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Dr. Khalid Islam, CEO of Gentium

Shares of Italy's Gentium plunged more than 40% this morning after the biotech announced a fresh round of woes with regulators--this time in Europe--for its vascular drug defibrotide. Back in 2011 Gentium yanked its FDA application after regulators raised a host of issues with its application for the drug. Today the biotech, which is listed on Nasdaq ($GENT), said that after its oral presentation in Europe it's expecting a negative opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use.

"While not a final decision, the company considers it unlikely that this position will change before the formal vote is undertaken next month," Gentium said in a statement. "If a formal negative recommendation is issued, and depending upon the nature of the objections, the company may appeal such negative decision."

The drug is designed to treat rare cases of veno-occlusive disease, a blockage of small veins that can occur after stem cell transplants. There is no approved therapy. And given Gentium's inability to make a case to regulators on either side of the Atlantic, there won't be anytime soon. 

"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," said Gentium CEO Dr. Khalid Islam. "We will work closely with the CHMP to address the Committee's concerns and remain confident of the favorable 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. We will continue to review all possible options with respect to next steps regarding the Company's MAA."

In the summer of 2011 Gentium said that the FDA raised questions about the "completeness of the datasets for both the treatment and prevention studies. The FDA requested that the company conduct additional quality reviews of the original datasets and databases. The FDA also requested additional details regarding the conduct and monitoring of the trials by the independent review committee."

- here's the press release
- read the Reuters story

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