Gentium S.p.A. (GENT) recently announced that it is seeking a re-examination of the negative opinion issued on defibrotide by the European Medicines Agency’s (:EMA) Committee for Medicinal Products for Human Use (CHMP).
The CHMP had issued the negative opinion in Mar 2013.
Gentium is looking to get the candidate approved for treating and preventing hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy. We note that currently there are no approved therapies to treat VOD in the US and EU.
We note that defibrotide was recommended by the European Group for Blood and Marrow Transplantation (EBMT) as the sole option to treat VOD patients. The EBMT has also expressed concern over the negative opinion issued by the CHMP regarding the approval of defibrotide in Europe.
The CHMP will review its initial opinion within 60 days and a final recommendation will available by the end of Jul 2013.
The regulatory proceedings for defibrotide were also far from smooth in the US. Defibrotide was granted fast-track designation by the US Food and Drug Administration (:FDA). In Jul 2011, a New Drug Application (:NDA) was filed with the FDA for defibrotide for the treatment of VOD.
As the FDA found many potential “Refuse to File” issues with the candidate, Gentium voluntarily withdrew its NDA in Aug 2011. Since then, Gentiumhas been working on it and expects to resubmit the NDA in 2013.
Gentium currently carries a Zacks Rank #3 (Hold).Currently, companies like Santarus, Inc. (SNTS), Jazz Pharmaceuticals (JAZZ) and Salix Pharmaceuticals Ltd. (SLXP) look well positioned with a Zacks Rank #1 (Strong Buy).
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