韩国GC绿十字公司血友病药物绿茵芷获批上市

2021-08-11 Jackzhao MedSci原创

8月10日,韩国GC绿十字公司宣布,中国国家药品监督管理局(NMPA)已正式批准了由其研发生产的绿茵芷(GreenGene F®; 通用名:注射用重组人凝血因子VIII)的上市申请,用于血友病

韩国GC绿十字与俄罗斯进行谈判,以生产新冠疫苗- 《韩国经济新闻》国际版

8月10日,韩国GC绿十字公司宣布,中国国家药品监督管理局(NMPA)已正式批准了由其研发生产的绿茵芷(GreenGene F®; 通用名:注射用重组人凝血因子VIII)的上市申请,用于血友病A患者(≧ 12周岁)的出血预防和控制治疗。

绿茵芷是第三代重组人凝血因子VIII。在生产全过程中,包括细胞培养、纯化、配置过程中未添加人或动物来源白蛋白,大大降低病原菌传播风险。生产过程中采用S/D法进行病毒灭活、采用纳米膜(20nm)的过滤技术用于去除病毒以及潜在的蛋白聚集体、使用了混合3种Amino-acids的高纯度产品的专利技术,保证产品质量,使其安全性显著提高。

PDF] Efficacy, Safety, and Pharmacokinetics of Beroctocog Alfa in Patients  Previously Treated for Hemophilia A | Semantic Scholar

Hyun, S. et al. “Efficacy, Safety, and Pharmacokinetics of Beroctocog Alfa in Patients Previously Treated for Hemophilia A.” Yonsei Medical Journal 56 (2015): 935 - 943.

此前,韩国一项前瞻性临床试验表明 12 岁以上的严重血友病 A 患者中评估 beroctocog alfa 的疗效、安全性和药代动力学。 材料和方法 70 名受试者接受 beroctocog alfa 作为急性出血的按需治疗。结果最终止血效果优35例(50%),良26例(37.1%)。该药物的总体有效率为 87.1%。大多数急性出血通过给予研究药物一次 (86.2%) 或两次 (10.0%) 治疗,每次输注的平均剂量为 28.55±6.53 IU/kg。 10名受试者共行12次手术,止血效果优7例(58.3%),良5例(41.7%),有效率100%。 88 名受试者中共有 52 名 (59.0%) 经历了 168 次不良事件。 11 名受试者发生 18 起严重不良事件 (10.7%),1 名受试者发生 2 起(轻度呼吸困难和面部水肿)与研究药物有关。只有一名受试者形成了新生因子 VIII 抑制剂,发生率为 1.4%(单侧 95% 置信上限:3.85%)。基线时和给药后 6 个月的最终消除半衰期分别为 13.3 小时和 12.6 小时。研究结论认为,beroctocog alfa 作为 A 型血友病患者急性出血的按需治疗或手术预防是安全有效的。

绿茵芷自2010年在韩国上市以来,来自真实世界的研究数据显示:绿茵芷能够安全、有效地预防和治疗血友病A。在2010年7月至2014年7月进行的上市后研究(PMS)显示:止血优良率91.3%,预防出血优良率89.4%。PMS研究结果表明绿茵芷安全可靠,对血友病A出血的预防和治疗有效。

中国医学科学院血液病医院血栓止血中心主任杨仁池教授表示,“对于血友病这种遗传性疾病而言,早诊断早治疗可以减少出血风险。尤其是对处于治疗黄金期的儿童和青少年患者,应该大力推行标准预防治疗,减少反复出血对关节的损伤,帮助患者和同龄人一起健康成长。近年来,我国血友病治疗取得了长足的进步,但由于血友病是需要终生治疗的疾病,在长期治疗过程中,治疗的安全、有效和经济可负担是广大血友病患者对于治疗的主要诉求。绿十字绿茵芷注射用重组人凝血因子VIII在海外上市多年,所积累的真实世界数据显示其安全有效。此次绿茵芷在中国的获批上市,将给我国血友病A患者提供更广泛的优质治疗选择。”

血友病A和B分别指因子Ⅷ和因子Ⅸ缺乏,由F8或F9基因的致病变异(如,突变、删除)引起,为X连锁出血性疾病,主要累及男性。血友病的遗传学特征会影响疾病的严重程度、抑制物的产生以及孕前检查和咨询。

血友病A是一种由凝血因子Ⅷ(Coagulation Factor Ⅷ,FⅧ)缺乏而导致的出血性疾病,是X染色体连锁的隐性遗传性疾病,主要由凝血因子Ⅷ基因突变引起。若反复出血不及时治疗,可导致关节畸形或假肿瘤形成,严重者可危及生命。目前,血友病A的主要治疗手段是凝血因子Ⅷ替代治疗,包括人凝血因子和重组人凝血因子。

血友病治疗中国指南(2020年版)指出血友病A的替代治疗首选基因重组FⅧ制剂或 病毒灭活的血源性FⅧ制剂,无上述条件时可选用 冷沉淀或新鲜冰冻血浆等。每输注1 IU/kg体重的 FⅧ可使体内FⅧ活性(FⅧ∶C)提高2%。FⅧ在体 内的半衰期8~12 h,要使体内FⅧ保持在一定水平 需每8~12 h输注1次。

参考资料;

Hyun, S. et al. “Efficacy, Safety, and Pharmacokinetics of Beroctocog Alfa in Patients Previously Treated for Hemophilia A.” Yonsei Medical Journal 56 (2015): 935 - 943.

 2020 Apr; 41(4): 265–271.Chinese. doi: 10.3760/cma.j.issn.0253-2727.2020.04.001

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在严重血友病A患者中,标准治疗是常规预防性,并静脉输注凝血因子VIII。原始出处:Midori Shima,Hideji Hanabusa,Masashi Taki,et al.Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,NEJM,2016.5.26