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Curr Top Med Chem. 2013;13(18):2386-94. doi: 10.2174/15680266113136660166.
Drug repurposing (drug repositioning, drug reprofiling, drug retasking) gains increasing importance as the development of new drugs becomes increasingly expensive. Though only a few compounds have been approved for new indications in the field of metabolic disorders, there are a number of substances which have the potential to become reprofiled in a new indication. Generally, reprofiled drugs for metabolic disorders can be classified in three groups. Group A contains those of which both, the original and repurposed indication, concern metabolic disorders. Group B comprises drugs, which were originally approved for non-metabolic disorders but show beneficial effects for metabolic disorders after repurposing. Group C comprises drugs, which were originally approved for metabolic disorders and are effective for non-metabolic disorders in their repurposed indication. Repurposed drugs in the field of metabolic disorders of group A include tetra-hydrobiopterin, originally indicated for phenylketonuria and now also approved for tetrahydrobiopterindeficiency, coenzyme-Q, originally approved for primary coenzyme-Q deficiency and reprofiled for statin-myopathy, and colesevelam, originally approved to reduce elevated low-density lipoprotein (LDL)-cholesterol (LDL-C) and now being approved for type-2-diabetes. An example of group C is phenylbutyrate, which was originally approved for urea-cycle disorders and meanwhile gained approval for progressive familial intrahepatic cholestasis type 2 due to mutations in the ABCB11 gene. Still additional compounds used to treat metabolic (non-metabolic) disorders show promising effects in non-metabolic (metabolic disorders) after repurposing in cell and tissue models. Future investigations will need to identify which candidate drugs may leave the pipeline status to acquire approval for new indications.
药物重定位（药物再定位、药物重新定位、药物重新任务）在开发新药物变得越来越昂贵的情况下变得越来越重要。尽管只有少数几种化合物在代谢紊乱领域获得了新适应症的批准，但有许多物质有可能在新适应症中重新定位。一般来说，代谢紊乱的再定位药物可以分为三组。A 组包含那些原始和重新定位的适应症都涉及代谢紊乱的药物。B 组包含最初批准用于非代谢紊乱但在重新定位后对代谢紊乱显示有益效果的药物。C 组包含最初批准用于代谢紊乱且在重新定位的适应症中对非代谢紊乱有效的药物。代谢紊乱领域的 A 组再定位药物包括四氢生物蝶呤，最初用于苯丙酮尿症，现在也批准用于四氢生物蝶呤缺乏症；辅酶 Q，最初批准用于原发性辅酶 Q 缺乏症，并重新用于他汀类药物肌病；考来烯胺，最初批准用于降低升高的低密度脂蛋白（LDL）-胆固醇（LDL-C），现在也批准用于 2 型糖尿病。C 组的一个例子是苯丁酸，它最初被批准用于尿素循环障碍，同时由于 ABCB11 基因突变，获得了进行性家族性肝内胆汁淤积症 2 型的批准。仍然有一些用于治疗代谢（非代谢）疾病的化合物在重新用于细胞和组织模型后在非代谢（代谢疾病）方面显示出有希望的效果。未来的研究将需要确定哪些候选药物可能会脱离管道状态，获得新适应症的批准。
