Department of Pharmacology, Faculty of Pharmacy, Ankara University, Ankara, Türkiye.
Department of Pharmacology, Faculty of Pharmacy, Izmir Katip Celebi University, Izmir, Türkiye.
Naunyn Schmiedebergs Arch Pharmacol. 2024 May;397(5):2949-2970. doi: 10.1007/s00210-024-03063-1. Epub 2024 Mar 26.
With 54 new drugs and seven cellular and gene therapy products, the approvals by the US Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels of 2020-2021. As in previous years of this annual review, we assign these new drugs to one of three levels of innovation: first drug against a condition ("first-in-indication"), first drug using a novel molecular mechanism ("first-in-class"), and "next-in-class," i.e., a drug using an already exploited molecular mechanism. We identify four (7%) "first-in-indication," 22 (36%) "first-in-class," and 35 (57%) "next-in-class" drugs. By treatment area, rare diseases (54%) and cancer drugs (23%) were once again the most prevalent (and partly overlapping) therapeutic areas. Other continuing trends were the use of accelerated regulatory approval pathways and the reliance on biopharmaceuticals (biologics). 2023 marks the approval of a first therapy based on CRISPR/Cas9 gene editing.
2023 年，美国食品和药物管理局 (FDA) 批准了 54 种新药物和 7 种细胞和基因治疗产品，从 2022 年的低谷恢复到 2020-2021 年的水平。与本年度审查的前几年一样，我们将这些新药分配到三个创新水平之一：针对一种病症的第一种药物（“同类首创”）、使用新分子机制的第一种药物（“同类首创”）和“同类次佳”，即使用已开发的分子机制的药物。我们确定了 4 种（7%）“同类首创”、22 种（36%）“同类首创”和 35 种（57%）“同类次佳”药物。按治疗领域划分，罕见病（54%）和癌症药物（23%）再次成为最常见的（部分重叠）治疗领域。其他持续的趋势是加速监管审批途径的使用和对生物制药（生物制剂）的依赖。2023 年标志着首次基于 CRISPR/Cas9 基因编辑的治疗方法获得批准。
