Wang Yingying, Yang Fuwei, Wang Baizhi, Xie Lijuan, Chen Wanying
Department of Neurology, China-Japan Union Hospital of Jilin University, Changchun, China.
Department of Neurosurgery, China-Japan Union Hospital of Jilin University, Changchun, China.
Eur J Med Chem. 2025 Mar 5;285:117241. doi: 10.1016/j.ejmech.2025.117241. Epub 2025 Jan 4.
In 2024, the U.S. Food and Drug Administration (FDA) has approved a range of new drugs, including both 32 new chemical entities (NCEs) and 18 biological entities (NBEs). Among the approved new drugs, small-molecule chemical drugs remained the main force for innovation, taking a commanding lead with a proportion of 64 %, covering targets like LACTB, PBP, THR-β, Raf, PDE3/4, and HIF. Monoclonal antibodies followed with 13 drugs (26 %), along with 2 protein-based drugs (4 %), 2 small nucleic acid drugs (4 %), and 1 parathyroid hormone analogue (2 %). The diseases treated by these approved new drugs were diverse, with the total number of new drugs for treating rare diseases and cancers ranking high. Additionally, multiple new drugs were also approved in the fields of anti-infective and central nervous system diseases. Similar to previous years, many of these drugs are likely to undergo accelerated approval processes to address urgent medical needs, particularly for rare diseases. This review provides an overview of the synthesis and clinical applications of NCEs approved by the FDA in 2024. The increasing importance of clinical applications has also been discussed. This review aims to provide valuable insights for the design of future drugs, particularly in the context of rare and complex diseases.
2024年，美国食品药品监督管理局（FDA）批准了一系列新药，包括32种新化学实体（NCEs）和18种生物实体（NBEs）。在获批的新药中，小分子化学药物仍是创新的主力军，占比64%，占据主导地位，涵盖LACTB、PBP、THR-β、Raf、PDE3/4和HIF等靶点。单克隆抗体紧随其后，有13种药物（占26%），还有2种基于蛋白质的药物（占4%）、2种小核酸药物（占4%）和1种甲状旁腺激素类似物（占2%）。这些获批新药治疗的疾病多种多样，治疗罕见病和癌症的新药总数排名靠前。此外，抗感染和中枢神经系统疾病领域也有多种新药获批。与往年类似，这些药物中的许多可能会经历加速审批程序，以满足迫切的医疗需求，尤其是针对罕见病。本综述概述了2024年FDA批准的新化学实体的合成及临床应用情况。还讨论了临床应用日益增长的重要性。本综述旨在为未来药物的设计提供有价值的见解，尤其是在罕见病和复杂疾病的背景下。
