2022年10月27日,协和麒麟宣布,惠尔金®(莫格利珠单抗)已获国家药品监督管理局(NMPA)的正式批准,用于治疗既往接受过系统性治疗的复发或难治性Sézary综合征(SS)或晚期(III/IV)蕈样肉芽肿(MF)成人患者。惠尔金®是中国首个也是唯一获批用于治疗SS和MF的靶向CCR4的生物制剂。莫格利珠单抗是一款同类首创的人源化单克隆抗体(mAb),靶向C-C趋化因子受体4(CCR4),后者是在SS和MF癌症细胞上存在持续表达的一种蛋白质。一旦惠尔金®与CCR4相结合,会在人体免疫系统中激活更多的免疫细胞,进而破坏癌细胞。
皮肤T细胞淋巴瘤(CTCL)属于罕见型非霍奇金淋巴瘤,SS和MF是CTCL的最常见的两种亚型,可累及患者皮肤、血液、淋巴结和内脏器官。CTCL较罕见,每10万人中约有24名患者。欧洲MF的年发病率约为1/11万~1/35万,而SS的年发病率为1/1000万。这两种亚型加起来约占所有CTCL病例的65%。患者普遍生活质量差、误诊率高且晚期缺乏靶向治疗药物,通常会出现皮损、发痒、疼痛以及不可预知的皮肤症状,并可能会导致进一步的并发症,从而影响他们的预期寿命。由于CTCL与湿疹及牛皮癣等常见皮肤病相似,患者平均需要2-7年才能得到确诊。晚期患者的预后明显更差,约半数患者(52%)的生存期仅为5年。
协和麒麟中国总经理李韵表示:“惠尔金®于2021年7月获得药品审评中心授予的优先审评资格,现已获国家药品监督管理局的正式批准。我们感谢监管机构对莫格利珠单抗的加速批准,这展示了政府为具有高度未被满足医疗需求的中国患者带来创新药物的决心。这也为中国的Sézary综合征和蕈样肉芽肿患者带来了希望,他们将能获得我们这款同类首创药物。协和麒麟通过追求生命科学和技术的进步来创造新的价值,我们致力于将更多创新性药物带入中国,从而造福广大中国患者。”
中国医学科学院肿瘤医院石远凯教授表示:“Sézary综合征和蕈样肉芽肿给患者的生活质量带来了沉重的负担。莫格利珠单抗的获批上市,将为中国这类患者带来一种全新作用机制的治疗选择。莫格利珠单抗改善了患者症状和生活质量,患者的皮肤病变和血液学指标对莫格利珠单抗治疗呈现出显著和持久的效果,中位无进展生存期显著延长。
此次获批是基于MAVORIC研究的结果。该研究是目前针对SS和MF患者最大规模的临床试验。研究结果表明,惠尔金®组患者的疾病控制时间是对照药物伏立诺他组患者的两倍多(中位无进展生存期:7.7个月 vs 3.1个月)。在处于各疾病阶段的MF/SS患者中,惠尔金®的应答率均高于伏立诺他*。惠尔金®和伏立诺他组患者的确认总缓解率分别为28%和5%(p<0.001)。惠尔金®的总体耐受性良好,安全性可控。
MAVORIC研究还评估了患者报告的症状体验和生活质量(QoL)。结果表明,与接受伏立诺他治疗的患者相比,接受惠尔金®的患者在症状、情绪、功能和整体生活质量方面均得到改善,体现在大部分功能和症状领域。总体而言,这些研究结果表明,与伏立诺他组相比,惠尔金®组患者的疾病及癌症特异性状况相关生活质量和总生活质量均得到改善,同时生活质量快速恶化的风险呈具有统计学意义的显著降低。
关于惠尔金®(莫格利珠单抗)
惠尔金®是一款同类首创人源化单克隆抗体(mAb),旨在与C-C趋化因子受体4(CCR4)相结合。惠尔金®与CCR4结合后,会在人体免疫系统中激活更多的免疫细胞,进而破坏癌细胞7。惠尔金®使用协和麒麟专有的POTELLIGENT®技术,可增强人体对治疗的自然免疫反应,从而提高对癌细胞的杀伤力。FDA已授予惠尔金®突破性疗法和孤儿药认定,。此外,惠尔金®还于2016年被欧盟委员会(EC)授予孤儿药认定。惠尔金®是首个也是唯一在中国和世界其他地区获批的靶向CCR4的生物制剂。
备注:*伏立诺他是美国FDA批准用于治疗蕈样肉芽肿和Sézary综合征的药物,目前尚未在中国获批
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