随着前沿生物技术的迅猛发展,以细胞和基因治疗药物为代表的前沿疗法药物(AdvancedTherapyMedicinalProducts,ATMPs)不断涌现。这类技术和产品给现阶段医学无法解决的疾病和人体功能障碍治疗带来了希望。由于前沿疗法药物活性成分的复杂性、异质性、与环境的相互作用和易降解性,其制剂的辅料(包材)选择给开发人员带来了巨大的挑战。这些挑战与ATMP质量、安全性和有效性息息相关。本文对ATMP细胞原料的稳定性问题和ATMP的生产特点进行讨论,并就目前美欧日市售的ATMP使用辅料(包材)的情况进行详细的回顾分析,最后提出了目前ATMP辅料(包材)开发的一些问题和建议。
<<With the rapid development of cutting-edge biotechnology,Advanced Therapy Medicinal Products (ATMPs),represented by cellular and gene therapy drugs,continue to emerge. Such technologies and products bring hope to the treatment of diseases and human dysfunctions that cannot be treated by current medicine. Due to the complexity,heterogeneity,interaction with the environment,and easy degradability of the active ingredients of ATMPs,the selection of excipients/packaging materials has brought great challenges to developers. These challenges are closely related to the quality,safety and effectiveness of ATMPs. This paper discusses the stability of ATMP cell raw materials and the production characteristics of ATMPs,and reviews the use of excipients/packaging materials in ATMPs currently sold in the United States,Europe and Japan,and finally proposes some questions and suggestions on the current development of ATMP excipients/packaging materials.
<<Keywords: | Advanced Therapy Medicinal Products (ATMPs)Cellular Therapy ProductsExcipientsPackaging MaterialsFormulations |