French foundation for rare diseases, 75014 Paris, France; Chemical and biological technologies for health unit (UTCBS), CNRS UMR8258, 75006 Paris, France; UTCBS, Inserm U1267, 75006 Paris, France; Faculté de pharmacie, université Paris Descartes, université de Paris, 75006 Paris, France.
French foundation for rare diseases, 75014 Paris, France.
Therapie. 2020 Apr;75(2):161-167. doi: 10.1016/j.therap.2020.02.007. Epub 2020 Feb 13.
While more than 7000 rare diseases have been identified, only about 5 percent benefit from a licensed treatment. As the majority of these diseases is life threatening, these facts underscore the need for new drugs. Drug repositioning is an alternative strategy in drug development, which represents an attractive opportunity for rare diseases. Drug repositioning (also called drug repurposing, drug reprofiling or drug re-tasking) consists in identifying for an already approved or investigational drug a new use outside the scope of the original medical indication. Drug repositioning is considered in the field of orphan drugs as being a faster and somehow less costly strategy than traditional new drug development for pharmaceutical companies. While several successful repositioning cases have been discovered by serendipity, most successes straightly derive from the molecular characterization of the concerned disease. This short commentary is mainly dedicated to these rationally-based success stories.
虽然已经确定了超过 7000 种罕见疾病，但只有约 5%的疾病受益于许可治疗。由于这些疾病大多数都有生命危险，因此这些事实突显了对新药的需求。药物重定位是药物开发的一种替代策略，它为罕见疾病提供了一个有吸引力的机会。药物重定位（也称为药物再利用、药物再定位或药物重新定向）是指为已经批准或正在研究的药物确定原医疗适应症范围之外的新用途。对于制药公司来说，药物重定位被认为是一种比传统新药开发更快且成本略低的策略。虽然有几个成功的药物重定位案例是偶然发现的，但大多数成功案例直接源于相关疾病的分子特征化。这篇简短的评论主要致力于这些基于理性的成功案例。
