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近期及预期的新型药物批准情况(2024年第二季度至2025年第一季度)

Recent and anticipated novel drug approvals (Q2 2024 through Q1 2025).

作者信息

Rim Matthew H, Karas Brittany L, Barada Farah, Dean Collin, Levitsky Andrew M

机构信息

Northwestern Medicine, Chicago, IL, USA.

IPD Analytics, Aventura, FL, USA.

出版信息

Am J Health Syst Pharm. 2024 Aug 12;81(16):733-738. doi: 10.1093/ajhp/zxae148.

Abstract

PURPOSE

Health-system pharmacists play a crucial role in monitoring the pharmaceutical pipeline to manage formularies, allocate resources, and optimize clinical programs for new therapies. This article aims to support pharmacists by providing periodic updates on new and anticipated novel drug approvals.

SUMMARY

Selected drug approvals anticipated in the 12-month period covering the second quarter of 2024 through the first quarter of 2025 are reviewed. The analysis emphasizes drugs expected to have significant clinical and financial impact in hospitals and clinics, as selected from 52 novel drugs awaiting US Food and Drug Administration approval. New cellular and gene therapies for cancers continued to strengthen the pipeline, in addition to new drugs targeting previously untreatable conditions. Several novel drugs are being developed for rare and ultra-rare diseases such as hemophilia, Niemann-Pick disease type C, hereditary angioedema, and aromatic l-amino acid decarboxylase deficiency.

CONCLUSION

The current drug pipeline includes new drugs with various indications for cancers and rare diseases as well as diabetes, acute coronary syndrome, chronic skin disorder, and chronic obstructive pulmonary disease.

摘要

目的

医疗系统药师在监测药品研发进程以管理药品目录、分配资源以及优化新疗法的临床项目方面发挥着关键作用。本文旨在通过定期更新新的和预期的新型药物批准情况来为药师提供支持。

总结

回顾了在2024年第二季度至2025年第一季度这12个月期间预计批准的部分药物。该分析重点关注从52种等待美国食品药品监督管理局批准的新型药物中挑选出的、预计会在医院和诊所产生重大临床和财务影响的药物。除了针对此前无法治疗病症的新药外,用于癌症的新型细胞和基因疗法继续充实着研发进程。正在为血友病、C型尼曼-匹克病、遗传性血管性水肿和芳香族L-氨基酸脱羧酶缺乏症等罕见和超罕见疾病研发几种新型药物。

结论

当前的药品研发进程包括针对癌症、罕见疾病以及糖尿病、急性冠状动脉综合征、慢性皮肤病和慢性阻塞性肺疾病等具有各种适应症的新药。

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