Gentium files for US approval of pig DNA product defibrotide in hepatic VOD
This article was originally published in Scrip
Executive Summary
Italian biopharmaceutical company Gentium has submitted an NDA to the US FDA for its lead compound, defibrotide, for the treatment of hepatic veno-occlusive disease (VOD) in adults and children undergoing haematopoietic stem cell transplantation (HSCT). The product, which is the sodium salt of a complex mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA, has been granted fast track status for the treatment of severe VOD in recipients of stem cell transplants by the US regulator and if approved, could become the first drug approved for this indication. Defibrotide also has orphan drug designation for severe VOD in this market.