Gentium jump on CHMP reversal for Defitelio

Shares of Gentium S.p.A. (NASDAQ:GENT) jumped $4.85 (50%) to $14.61 on Friday after EMA's CHMP backed approval of Defitelio defibrotide to treat severe hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation therapy, reversing a prior negative recommendation. The committee said Defitelio should be approved under EMA's exceptional circumstances clause. In March, CHMP recommended against approval of Defitelio to both treat and prevent VOD. Gentium requested a reexamination of the negative opinion and submitted a revised MAA that focused on treating severe VOD and preventing VOD in high-risk patients. The company subsequently withdrew the prevention indication. In March, CHMP had said data from a prevention study did not provide "sufficiently convincing evidence" of Defitelio's benefit (see BioCentury Extra, March 22).

CHMP examined additional data submitted by Gentium from a U.S. patient registry and concluded the benefits of Defitelio outweigh its risks. The data showed patients with VOD who received Defitelio plus standard of care had a higher survival rate 100 days post-transplantation compared with patients who received standard of care alone. ...