CHMP recommends Defitelio (Gentium) for Veno Occlusive Disease
On 25 July 2013, the CHMP, following a re-examination procedure, adopted a final positive opinion, recommending to grant a marketing authorisation for the medicinal product Defitelio from Gentium, 80 mg/mL concentrate for solution for infusion intended for the treatment of severe hepatic Veno-Occlusive Disease (VOD) also known as Sinusoidal Obstructive Syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.
Defitelio was designated as an orphan medicinal product on 29 July 2004. The active substance of Defitelio is defibrotide, a drug for the treatment of severe veno-occlusive disease (VOD) in patients undergoing haematopoietic (blood) stem-cell (B01AX01). The mechanism of action of defibrotide has not been fully elucidated.
Defibrotide was rejected by the FDA in March 2013.
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