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药理学的一年:2024年美国食品药品监督管理局批准的新药

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2024.

作者信息

Aksoyalp Zinnet Sevval, Kayki-Mutlu Gizem, Wojnowski Leszek, Michel Martin C

机构信息

Department of Pharmacology, Faculty of Pharmacy, Izmir Katip Celebi University, Izmir, Turkey.

Department of Pharmacology, Faculty of Pharmacy, Ankara University, Ankara, Turkey.

出版信息

Naunyn Schmiedebergs Arch Pharmacol. 2025 May;398(5):5077-5099. doi: 10.1007/s00210-025-04020-2. Epub 2025 Mar 31.

Abstract

The US Food and Drug Administration approved 50 new drugs and nine new cellular and gene therapy products in 2024, i.e., a total of 59 new medical therapies. The latter group represented three treatments each for oncology and hematology/immunotherapy, and one each for neurology, genetic disorders, and cardiovascular disorders. Oncology, hematology/immunotherapy, and neurological disorders (14, six, and seven, respectively) also were highly prevalent among classic medications. Looking at trends over the past 5 years, we observe a greater share in first-in-class medications, more fast-track approvals, and mRNA/gene/cell-based therapies. While small molecules remain the largest fraction, their percentage has been declining substantially over the past 5 years. Taking together, these findings testify to the commitment of the pharmaceutical industry for innovative treatments, including conditions for which no approved therapies existed. On the other hand, there also is a trend for approvals for narrowly focused conditions such as tumors defined by genetic alterations.

摘要

2024年,美国食品药品监督管理局批准了50种新药和9种新的细胞与基因疗法产品,即总共59种新的医学疗法。后一组包括肿瘤学和血液学/免疫疗法各三种治疗方法,以及神经病学、遗传性疾病和心血管疾病各一种治疗方法。肿瘤学、血液学/免疫疗法和神经系统疾病(分别为14种、6种和7种)在传统药物中也非常普遍。回顾过去5年的趋势,我们观察到同类首创药物的份额更大、快速通道批准更多,以及基于mRNA/基因/细胞的疗法。虽然小分子药物仍然占最大比例,但在过去5年中其百分比一直在大幅下降。综上所述,这些发现证明了制药行业对创新治疗的承诺,包括针对尚无批准疗法的病症。另一方面,对于如由基因改变定义的肿瘤等针对性狭窄的病症,也存在批准的趋势。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a46c/11985671/6017e7e19537/210_2025_4020_Fig1_HTML.jpg

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