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2023年美国食品药品监督管理局肽和寡核苷酸采集活动

2023 FDA TIDES (Peptides and Oligonucleotides) Harvest.

作者信息

Al Shaer Danah, Al Musaimi Othman, Albericio Fernando, de la Torre Beatriz G

机构信息

Department of Medicinal Chemistry, Evotec (UK) Ltd., Abingdon OX14 4R, UK.

School of Pharmacy, Faculty of Medical Sciences, Newcastle upon Tyne NE1 7RU, UK.

出版信息

Pharmaceuticals (Basel). 2024 Feb 13;17(2):243. doi: 10.3390/ph17020243.

Abstract

A total of nine TIDES (pepTIDES and oligonucleoTIDES) were approved by the FDA during 2023. The four approved oligonucleotides are indicated for various types of disorders, including amyotrophic lateral sclerosis, geographic atrophy, primary hyperoxaluria type 1, and polyneuropathy of hereditary transthyretin-mediated amyloidosis. All oligonucleotides show chemically modified structures to enhance their stability and therapeutic effectiveness as antisense or aptamer oligomers. Some of them demonstrate various types of conjugation to driving ligands. The approved peptides comprise various structures, including linear, cyclic, and lipopeptides, and have diverse applications. Interestingly, the FDA has granted its first orphan drug designation for a peptide-based drug as a highly selective chemokine antagonist. Furthermore, Rett syndrome has found its first-ever core symptoms treatment, which is also peptide-based. Here, we analyze the TIDES approved in 2023 on the basis of their chemical structure, medical target, mode of action, administration route, and common adverse effects.

摘要

2023年期间,美国食品药品监督管理局(FDA)总共批准了九种TIDES(肽类和寡核苷酸类)药物。四种获批的寡核苷酸适用于多种类型的疾病,包括肌萎缩侧索硬化症、地图样萎缩、1型原发性高草酸尿症以及遗传性转甲状腺素蛋白介导的淀粉样变性多神经病。所有寡核苷酸均显示出化学修饰结构,以增强其作为反义或适体寡聚物的稳定性和治疗效果。其中一些还展示了与驱动配体的各种类型的偶联。获批的肽类药物具有多种结构,包括线性、环状和脂肽,并具有多种应用。有趣的是,FDA首次授予一种基于肽的药物孤儿药认定,该药物为一种高度选择性的趋化因子拮抗剂。此外,雷特综合征也首次有了基于肽的核心症状治疗方法。在此,我们根据2023年获批的TIDES药物的化学结构、医学靶点、作用方式、给药途径和常见不良反应进行分析。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6877/10893093/79c9160b3c49/pharmaceuticals-17-00243-g001.jpg

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