Italian drugmaker Gentium S.p.A. (GENT) announced Friday that it has received negative opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use or CHMP, on Defibrotide for the treatment and prevention of hepatic veno-occlusive disease or VOD, in adults and children undergoing blood stem cell transplantation therapy.
The CHMP has recommended the refusal of the marketing authorization for Defibrotide as it concluded that the benefits of the drug did not outweigh its risks.
Meanwhile, Gentium said it intends to appeal the negative opinion issued by the CHMP as it remains convinced of the favorable benefit/risk profile of Defibrotide. The company will also request a re-examination of the CHMP opinion within 15 days.
Defibrotide is the only option recommended by the European Group for Blood and Marrow Transplantation or EBMT, for the treatment VOD, an unmet medical need where no agents are currently approved.
Separately, the EBMT expressed its concern at the decision of the CHMP not to recommend a Marketing Authorization for Defibrotide for the treatment and prevention of VOD. The EBMT is the largest professional body in the field of Haematopoietic Stem Cell transplantation or HSCT, with more than 4,200 members in 570 transplant centers in 57 countries.
VOD is a rare but serious condition in which the veins in the liver become blocked or damaged by the high doses of chemotherapy and radiation as cancer treatment, leading to liver dysfunction. Defibrotide was designated an 'orphan medicine', a medicine for rare diseases, in July 2004 for the treatment and prevention of hepatic VOD.
VOD affects around 3500 to 4000 patients in the EU, but unfortunately 800 to 1000 of these patients will develop severe VOD which is characterized by liver and other organ failure. Severe VOD carries a mortality rate of between 70 to 90 percent.
Currently, there are no approved treatments for VOD by either the U.S. Food and Drug Administration or the European Medicines Agency.
However, EU patients will continue to have access to Defibrotide, under a named patient program, where available. More than 230 of 524 EU institutions performing HSCT use this drug on a Named Patient basis.
Gentium's Marketing Authorization Application for Defibrotide was submitted to the European Medicines Agency, or EMA, in May 2011, and the CHMP issued a LoQs (List of Questions) in September 2011, seeking some clarifications from the company regarding the drug candidate.
In February 2012, Gentium responded to the EMA's LoQs 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 2012.
In line with the regulatory timetable, the company submitted its response to the LoOI received from the CHMP in June 2012. As part of the review process, the company has also presented oral explanations on September 19, 2012 to the EMA's CHMP regarding the Defibrotide application.
Earlier in August 2011, Gentium voluntarily withdrew the Defibrotide new drug application or NDA, submitted to the FDA seeking approval for a similar use after concerns were raised regarding the completeness of the datasets for both the treatment and prevention studies. The company is expected to re-submit the NDA in the first half of 2013 to the FDA.
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.
In Friday's regular trading session, GENT is currently trading at $7.62, down $0.51 or 6.27% on a volume of 4,500 shares.
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