--诺和诺德（Novo Nordisk）宣布，重组凝血因子VIII产品NovoEight（turoctocog alpha）生物制品许可申请（BLA）获FDA批准。FDA批准NovoEight用于A型血友病成人和儿童患者：（1）出血事件的预防和治疗；（2）围手术期管理；（3）常规预防措施，以防止或减少出血发作的频率。
NovoEight是turoctocog alpha的商品名，该药是诺德诺德第三代重组凝血因子VIII，利用了最新的基因重组及蛋白纯化技术，用于A型血友病患者出血事件的预防和治疗。
NovoEight已在III期Guardian项目中进行了评价，这是迄今为止在A型血友病患者中开展的最大的临床预注册试验项目，涉及超过200例A型血友病患者。
这些III期临床试验包括既往接受过治疗的重度A型血友病成人及儿童患者，研究结果证明了turoctocog-alpha对出血事件的预防及治疗疗效，同时患者体内未产生抑制剂。
今年9月，NovoEight也获得了欧洲药品管理局（EMA）人用医药产品委员会（CHMP）建议批准的积极意见。欧盟委员会（EC）预计将在未来几个月内做出审查决定。
诺和诺德也已向其他国家监管部门提交了turoctocog-alpha的监管文件。
英文原文：Novoeight® (turoctocog alfa) receives approval from the FDA
Bagsværd, Denmark, 16 October 2013 – Today, Novo Nordisk announced that the U.S. Food and Drug Administration (FDA) has approved its Biologics License Application (BLA) for recombinant coagulation factor VIII, Novoeight®.
The FDA approved Novoeight® for use in adults and children with haemophilia A for:
- Control and prevention of bleeding
- Perioperative management
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
Novoeight® has been studied in the guardian™ clinical programme; one of the largest and most comprehensive pre-registration clinical trial programmes in the field of haemophilia therapy with more than 210 severe haemophilia A patients. In the completed trials Novoeight® demonstrated good efficacy in preventing and treating bleeds and had no confirmed inhibitor development, and all patients in the surgery trial were treated effectively. Novoeight® will be launched with the newly introduced prefilled device, MixPro®.
“The approval of Novoeight® marks an important step in offering a new alternative for people with haemophilia A, and demonstrates our commitment to haemophilia,” says Mads Krogsgaard Thomsen, executive vice president and chief science officer at Novo Nordisk.
Awaiting the expiration of existing patents, Novo Nordisk plans to launch in the U.S. Novoeight® shortly after April 2015.
Novoeight® received positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in September 2013. Applications for regulatory approval have also been submitted in a range of other countries.
About haemophilia A
Haemophilia is a chronic, inherited bleeding disorder that primarily affects males. People with haemophilia A are either missing or have a malfunctioning factor VIII protein, which is essential for proper blood clotting. People with haemophilia A have a tendency to bleed longer than most, or to bleed internally, because they are missing this clotting factor. To manage the disease and stop bleeding, people with haemophilia A must replace the missing factor VIII protein through intravenous injection of the clotting factor.
Globally it is estimated that 350,000 people have haemophilia A. The disease is severely under-diagnosed in developing countries.