首页 » FDA信息 » 血液病巨头百特(Baxter)向FDA提交长效版ADVATE监管申请


来源:生物谷 2014-12-02 14:06

2014年12月2日讯 /生物谷BIOON/ --血液病巨头百特国际(Baxter International)近日宣布,已向FDA提交了BAX 855的生物制品许可申请(BLA)。BAX 855是基于已上市产品ADVATE开发的一种半衰期延长的重组凝血因子VIII(rFVIII),该药开发用于A型血有病的治疗。ADVATE是百特的龙头产品,上市时间超过10年,是全球处方量最多的FVIII产品。BAX 855是ADVATE的长效版。如果获批,BAX 855将成为12岁及以上A型血友病患者一种重要的新治疗选择。(相关阅读:猪凝血因子造福人类——FDA批准百特血友病新药Obizur

BAX 855 BLA的提交,是基于一项前瞻性全球性III期临床试验的结果,该研究在137例既往治疗过的A型血友病患者中开展,数据表明,BAX 855达到了控制和预防出血事件及日常预防的主要终点。与按需治疗相比,每2周注射一次的预防性治疗方案,使平均年出血率降低了95%。

百特计划在未来几个月内提交BAX 855关键临床试验的更多数据。一旦儿科临床试验完成,百特预计将在2016年向欧洲药品管理局(EMA)提交BAX 855的上市许可申请(MAA)。

BAX 855是百特龙头产品血液疾病药物Advate的增强版。ADVATE是一种全长重组FVIII产品,未添加任何血液添加剂,适用于A型血友病患者出血的治疗和预防,不适用于血管性血友病(von Willebrand disease,vWD)的治疗,该药已获全球64个国家批准。BAX 855利用了专有的聚乙二醇化(PEGylation)技术,旨在延长蛋白在体内的活性持续时间。该专有技术已应用于多种已上市药物。

A 型血友病,又称典型血友病,是一种由于缺乏凝血因子 VIII 或凝血因子 VIII 蛋白缺陷而导致的遗传病。

今年6月,FDA 已批准了其潜在竞争对手百健艾迪(Biogen Idec)生产的长效血友病药物 Eloctate ,该药首个具有延长的循环半衰期的重组凝血因子VIII产品,该药的适应症为:用于儿童和成人A型血友病患者出血事件的控制和预防,围术期管理和常规预防。(相关阅读:百健艾迪A型血友病新药Eloctate获FDA批准


英文原文:Baxter Submits Application for U.S. FDA Approval of BAX 855, Extended Half-Life Recombinant FVIII Based on ADVATE for Hemophilia A

Twice-Weekly Prophylactic Regimen Resulted in 95% Reduction in Median Annualized Bleed Rate Compared to On-Demand Treatment

DEERFIELD, Ill., December 1, 2014 - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) to the United States Food and Drug Administration (FDA) for the approval of BAX 855, an investigational, extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic Factor (Recombinant)].

"The evidence from our late-stage controlled trial showcases that BAX 855 could become an important new treatment option for people with hemophilia A, expanding on more than a decade of experience with ADVATE," said John Orloff, M.D., vice president and global head of research and development for Baxter BioScience. "BAX 855 represents our continued commitment to developing valuable new options for the hemophilia community that address the needs of patients around the world."

The submission is based on positive results from a prospective, global, multi-center, open-label, two-arm Phase 3 study of 137 previously treated patients (PTP). The results demonstrated that BAX 855 met its primary endpoint in the control and prevention of bleeding episodes and routine prophylaxis for patients who were 12 years or older.

Patients in a twice-weekly prophylaxis arm experienced a 95 percent reduction in median ABR as compared to those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating bleeding episodes, 96 percent of which were controlled with one or two infusions. No patients developed inhibitors to BAX 855 and no treatment-related serious adverse events, including hypersensitivity, were reported. The most common (three patients) product-related adverse reaction was headache.

Baxter expects to present additional data from the BAX 855 pivotal trial in the coming months. The continuation study remains ongoing for patients who completed the pivotal trial and the Phase 3 study among previously treated patients under the age of 12 with severe hemophilia A is progressing. Once the pediatric study is complete, Baxter expects to file for marketing authorization with the European Medicines Agency in 2016. Upon approval, BAX 855 is expected to be produced at the company's Singapore manufacturing facility, one of several sites currently approved for ADVATE production.

BAX 855 is based on ADVATE, a full-length FVIII molecule with more than 10 years of real-world experience. Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR), BAX 855 leverages proprietary PEGylation technology designed to extend the duration of activity of proteins in the body. This proprietary technology has been used for over 10 years in a number of approved medicines that treat chronic or serious conditions.

ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency or classic hemophilia) for:

Control and prevention of bleeding episodes.
Perioperative management.
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

ADVATE is not indicated for the treatment of von Willebrand disease.

ADVATE has a demonstrated efficacy and safety profile. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated.

There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.

ADVATE is the world's most prescribed FVIII treatment. It is currently approved in 64 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

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