新功能、新界面、新体验,扫描即可下载生物谷APP!
首页 » FDA信息 » 猪凝血因子造福人类——FDA批准百特血友病新药Obizur

猪凝血因子造福人类——FDA批准百特血友病新药Obizur

来源:生物谷 2014-10-28 09:18

2014年10月28日讯 /生物谷BIOON/ --导读:目前,百特(Baxter)正在对旗下生物制药(Biopharmaceutical)与医疗技术业务进行拆分,拆分出的生物制药公司名为Baxalta,并计划于2015年中期在纽交所独立上市。Baxalta这一名称由Baxter与“alta”组合而成,“alta”由拉丁语“altus”衍生而来,寓意“高”或“深刻”,Baxalta将专注于血友病药物、免疫肿瘤产品及疫苗,并已启动基因治疗项目同时计划进入生物仿制药领域。而百特(Baxter)将专注于药物输送系统及透析和医院产品。

近日,Baxalta血友病专营权进一步得到扩大。新药Obizur获得FDA批准,用于获得性血友病A(AHA)成人患者的治疗。Obizur是FDA批准用于AHA治疗的首个重组猪凝血因子VIII(FVIII)产品。AHA是一种非常罕见的急性出血性疾病,发病率为百万分之一。

百特(Baxter)近日宣布,FDA已批准Obizur[抗血友病因子(重组),猪序列]用于获得性血友病A(acquired hemophilia A,AHA)成人患者出血发作(bleeding episodes)的治疗。获得性血友病A(AHA)是一种非常罕见、可能危及生命的急性出血性疾病,是以循环血中出现抗凝血因子VIII(FVIII)的自身抗体为特征的一种自身免疫性疾病,其特点为既往无出血史和无阳性家族史的患者出现自发性出血或者在手术、外伤或侵入性检查时发生异常出血。此前,FDA已授予Obizur孤儿药地位及优先审查资格,获得性血友病A(AHA)的发病率为百万分之一。

Obizur是FDA批准用于获得性血友病A(AHA)的首个重组猪FVIII药物,使医生除了临床评估外通过评估因子VIII的活动水平管理治疗的有效性和安全性。Obizur的设计原理是,在获得性血友病A(AHA)患者中,血液循环中存在的抗人FVIII自身抗体能够结合人FVIII,而猪FVIII不易受到抗人FVIII自身抗体的灭活,因此重组猪FVIII可替代人FVIII发挥作用。

Obizur的获批,是基于一项全球性、前瞻性、对照多中心II/III期开放标签研究的积极数据,该研究评估了Obizur治疗获得性血友病A(AHA)成人患者严重出血发作的疗效和安全性。研究中,28例患者参与了疗效评估,数据表明,在Obizur初始输液后24小时,所有患者(28/28)均表现出积极的反应,意味着出血有效或部分有效停止及临床症状的显著改善,共有86%(24/28)患者的初次出血发作得到成功治疗。此外,有29例患者参与安全性评估,数据表明,有略大于5%的患者产生抗猪FVIII抗体。

百特计划在未来几个月内在美国推出Obizur,目前,欧盟和加拿大监管机构也正在审查Obizur。(生物谷Bioon.com)

英文原文:FDA Approves Baxter's OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence], for Acquired Hemophilia A

DEERFIELD, Ill.--Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) has approved OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A (AHA), a very rare and potentially life-threatening acute bleeding disorder. OBIZUR was granted orphan-drug status by the FDA and its review was prioritized based on AHA's classification as a rare disease and the potential for the treatment to address an important unmet medical need.

OBIZUR is the first recombinant porcine FVIII treatment approved for AHA that allows physicians to manage the treatment's efficacy and safety by measuring factor VIII activity levels in addition to clinical assessments. OBIZUR replaces the inhibited human factor VIII with a recombinant porcine sequence factor VIII based on the rationale that it is less susceptible to inactivation by circulating human factor VIII antibodies.

''The approval of OBIZUR is welcome news for the hemophilia community based on the data from the first clinical trial designed specifically for acquired hemophilia A, which found that all patients responded to treatment within 24 hours,'' said Dr. Rebecca Kruse-Jarres, Director of the Hemophilia Care Program at Puget Sound Blood Center in Seattle and the clinical trial's principal investigator. ''Importantly, this new option to treat bleeding episodes will enable us to measure factor VIII levels, thus giving us an objective marker of hemostasis that can guide dosing and prevent overdosing.''

The approval is based on a global, prospective, controlled, multi-center Phase 2/3 open-label clinical trial that examined the efficacy of OBIZUR in the treatment of serious bleeding episodes in adults with AHA (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with OBIZUR (28/28) showed a positive response, meaning an effective or partially effective response with bleeding stopped or reduced and clinical improvement, at 24 hours after the initial infusion. A total of 86 percent (24/28) had successful treatment of the initial bleeding episode. The overall treatment success was determined by the investigator based on the ability to discontinue or reduce the dose and/or dosing frequency of OBIZUR. Common adverse reactions observed in greater than five percent of 29 patients in the clinical trial were development of inhibitors to porcine FVIII.

''As a new treatment option with the ability to measure FVIII activity in the body, OBIZUR will address important unmet needs for patients with acquired hemophilia A, a potentially life-threatening condition,'' said Brian Goff, head of Baxter's hemophilia franchise. ''This approval reflects Baxter's long-standing commitment to discovering new options for hemophilia patients and adds to our portfolio of treatments that reduce the burden of these diseases.''

OBIZUR will be commercially available in the United States in the coming months and is currently under regulatory review in Europe and Canada.

About OBIZUR

OBIZUR [Antihemophilic Factor (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in adults with acquired hemophilia A.

Limitations of Use:

Safety and Efficacy of OBIZUR has not been established in patients with baseline anti-porcine factor VIII inhibitor titer greater than 20 BU.

OBIZUR is not indicated for the treatment of congenital hemophilia A or von Willebrand disease.

温馨提示:87%用户都在生物谷APP上阅读,扫描立刻下载! 天天精彩!


相关标签

最新会议 培训班 期刊库