Abstract：
In 2023, seventy novel drugs received market authorization for the first time in either Europe (by the EMA and the MHRA) or in the United States (by the FDA). Confirming a steady recent trend, more than half of these drugs target rare diseases or intractable forms of cancer. Thirty drugs are categorized as \"first-in-class\" (FIC), illustrating the quality of research and innovation that drives new chemical entity discovery and development. We succinctly describe the mechanism of action of most of these FIC drugs and discuss the therapeutic areas covered, as well as the chemical category to which these drugs belong. The 2023 novel drug list also demonstrates an unabated emphasis on polypeptides (recombinant proteins and antibodies), Advanced Therapy Medicinal Products (gene and cell therapies) and RNA therapeutics, including the first-ever approval of a CRISPR-Cas9-based gene-editing cell therapy.
摘要：
2023年，70种新药首次在欧洲（EMA和MHRA）或美国（FDA）获得市场授权。证实了近期的稳定趋势，这些药物中有一半以上针对罕见疾病或顽固性癌症。30种药物被归类为“一流”(FIC)，说明推动新化学实体发现和发展的研究和创新的质量。我们简洁地描述了大多数FIC药物的作用机制，并讨论了所涵盖的治疗领域，以及这些药物所属的化学类别。2023年的新药清单也显示出对多肽（重组蛋白和抗体）的重视程度不减，高级治疗药物产品（基因和细胞疗法）和RNA疗法，包括首次批准基于CRISPR-Cas9的基因编辑细胞疗法。
