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news.Gentium has said that the FDA has granted orphan drug designation to Defibrotide for the prevention of hepatic veno-occlusive disease in the US.
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Veno-occlusive disease (VOD) occurs when certain high dose chemotherapy and radiation therapies and stem cell transplantation damage cells of the blood vessels which results in a blockage of the small veins of the liver.
“Previous studies have demonstrated compelling clinical results using Defibrotide to prevent VOD. We are currently conducting a phase II/III trial of Defibrotide to prevent VOD in pediatric patients in Europe, and are looking forward to initiating a US and European phase II/III study of Defibrotide to prevent VOD in adults in the coming months,” said Laura Ferro, president and CEO of Gentium.
Orphan drug designation is given to products for rare diseases. Orphan drug designation may qualify recipients for exclusive marketing rights in the US for seven years. The designation also positions Gentium to potentially benefit from certain tax credits.