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畅销(2026)小分子孤儿药:探寻其化学本质。

Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry.

机构信息

Group of Catalysis, Synthesis and Organic Green Chemistry, Department of Pharmaceutical Sciences, University of Perugia, Via del Liceo 1, 06123 Perugia, Italy.

出版信息

Int J Mol Sci. 2023 Jan 4;24(2):930. doi: 10.3390/ijms24020930.

DOI:10.3390/ijms24020930
PMID:36674441
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9864910/
Abstract

This review describes, from a chemical point of view, the top "blockbuster" small molecule orphan drugs according to their forecasted sales in 2026. Orphan drugs are intended for the treatment, prevention, or diagnosis of a rare disease or condition. These molecules are mostly addressed to the treatment of rare forms of cancer. The respiratory and central nervous systems represent other common therapeutic subcategories. This work will show how the orphan drugs market has significantly grown and will account for a consistent part of prescriptions by 2026.

摘要

这篇综述从化学角度描述了根据预测销售额排名前 2026 位的“重磅炸弹”孤儿小分子药物。孤儿药旨在治疗、预防或诊断罕见疾病或病症。这些药物主要用于治疗罕见形式的癌症。呼吸系统和中枢神经系统是其他常见的治疗亚类。本文将展示孤儿药市场如何显著增长,并在 2026 年占据处方的重要部分。

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