Defitelio®（去纤）严重肝静脉闭塞症（“VOD”）的成人和儿童接受造血干细胞移植疗法的治疗。 Defitelio®是欧盟第一个批准用于治疗这种危及生命的疾病。此授权是来自欧洲医药局（“EMA”）接受了委员会的人用药品的产品（“CHMP”）的积极意见7月26日正式批准，2013年本公司拟开始在欧盟Defitelio®商业化在2013年12月。
“Defitelio®今天的授权代表万民法的一个重要里程碑，”哈立德伊斯兰万民法SpA的博士，董事长兼首席执行官说：“作为第一种药物被批准用于重度VOD的治疗，Defitelio®会为医师提供一个救命选项寻址疾病具有高死亡率高的未满足的医疗需求，超过80％。万民法是这样的成就，我们相信这将提供舒适和希望病人和家属感到骄傲。“
“这个决定让万民法到市场Defitelio®在欧盟的28个会员国。我们希望通过我们自己的销售队伍，并通过我们的分销商合作伙伴在2013年底开始Defitelio®的商业销售，”阿德里安·黑格，商务高级副总裁说：操作和万民法有限公司的首席运营官。
关于VOD
静脉闭塞性疾病（VOD）是一种潜在的威胁生命的情况，这通常会发生作为干细胞移植的显著并发症。用作干细胞移植的一部分可损害肝脏血管衬里的细胞，并导致在视频点播，在这导致肝衰竭，并可能导致在其他器官显著功能障碍肝小静脉阻塞某些高剂量预处理方案如肾脏和肺（所谓严重VOD）。干细胞移植是一种常用的治疗方式以下高剂量化疗和放射治疗的血液肿瘤和在成人和儿童的其他条件。目前还没有批准的代理VOD在美国或欧盟的治疗或预防。
Information
Generic Name: defibrotide  
Trade Name: Defitelio  
Synonym: Noravid, Prociclide 
Entry Type: New molecular entity  
Development and Regulatory status
UK: Launched 
EU: Launched 
US: Pre-registration (Filed) 
UK launch Plans: Available only to registered users
Actual UK launch date: June 2014 
Comments
Dec 14: New Drug Application has been submitted to the FDA for defibrotide for the treatment of severe hepatic veno-occlusive disease (VOD) in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy. Defibrotide has been granted Fast Track Designation to treat severe VOD by the FDA. [27]
15/12/2014 09:46:26 
Jun 14: Launched in the UK [26].
09/06/2014 15:53:18 
Mar 14: Launched in Germany & Austria. Jazz Pharmaceuticals, which gained ownership of Defitelio via its purchase of Gentium earlier in 2014, expects to continue launch in 27 additional European countries on a rolling basis during 2014 & 2015 [25].
15/04/2014 17:22:31 
Oct 13: European Commission has granted a Marketing Authorization for Defitelio® (defibrotide) for the treatment of severe hepatic veno-occlusive disease in adults and children undergoing hematopoietic stem cell transplantation therapy. [23]
23/10/2013 16:47:45 
Jul 13: EU positive opinion for Defitelio to be used for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants aged 1 month to 18 years. After re-examining their original negative opinion, the CHMP concluded that, in view of results from a US patient registry showing pts with severe VOD who received Defitelio plus standard care had better outcomes, including a higher survival rate after 100 days following transplantation, than those given standard care alone, along with other available data, the benefits of Defitelio outweigh its risks. A risk management plan will be put in place & the applicant will be required to provide further data on the medicine through a registry to be set up in the EU. The CHMP recommended that Defitelio be authorised in the EU under ‘exceptional circumstances’ [22].
26/07/2013 14:26:49 
Jun 13: The Company has filed the documentation requesting a re-examination of the negative opinion given by the CHMP. A final recommendation