CAR T-cell product performance in haematological malignancies before and after marketing authorisation.
CAR T-cell product performance in haematological malignancies before and after marketing authorisation.
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DOI：
10.1016/s1470-2045(19)30729-6
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发表时间：
2020-02
期刊：
影响因子：
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通讯作者：
Abou-El-Enein M
中科院分区：
文献类型：
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作者：
Elsallab M;Levine BL;Wayne AS;Abou-El-Enein M
嵌合抗原受体（CAR）T细胞代表了治疗血液恶性肿瘤的有效新方法。两种CAR T细胞疗法，tisagenlecleucel和axicabtagene ciloleucel，已在欧洲和美国以及其他几个国家获得批准，用于治疗白血病和淋巴瘤。这些批准标志着细胞和基因治疗领域的一个重要里程碑。然而，这些创新疗法的临床开发和监管评估面临着一些挑战，这些挑战被认为是未来类似产品的重要经验教训。在此，我们检查了产品的非临床和临床数据包，以概述在欧洲监管评估过程中遇到的挑战，并提供授权后性能的最新信息。
Chimeric antigen receptor (CAR) T cells represent a potent new approach to treat haematological malignancies. Two CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel, have been approved in Europe and the USA, as well as several other countries, for the treatment of leukaemia and lymphoma. These approvals marked a major milestone in the field of cell and gene therapies. However, the clinical development and regulatory evaluation of these innovative therapies faced several challenges that are considered important lessons learned for future similar products. Here, we examine the products’ non-clinical and clinical data packages to outline the challenges encountered during the regulatory evaluation process in Europe, and to provide an update on their performance after authorisation.