Kinch Michael S, Umlauf Sheila, Plummer Mark
Yale Center for Molecular Discovery, West Haven, CT 06516, USA.
Yale Center for Molecular Discovery, West Haven, CT 06516, USA.
Drug Discov Today. 2015 Jun;20(6):648-51. doi: 10.1016/j.drudis.2015.02.002. Epub 2015 Feb 11.
Metabolic diseases encompass a constellation of maladies including obesity and diabetes that are among the fastest growing epidemics throughout the world. An analysis of new molecular entities (NMEs) targeting metabolic diseases reveals the rate of approval for new drugs increased in the mid-1990s and now stands at approximately two per year. The increase is largely attributed to a recent emphasis on treatments for inborn errors of metabolism. In particular, biotechnology companies have focused on rare genetic disorders, which are often treated with biologic-based NMEs that target novel pathways and qualify for orphan drug status. By contrast, NME development by pharmaceutical companies tended toward conventional small molecular targeting of nongenetic disorders such as diabetes.
代谢性疾病包括一系列疾病，如肥胖症和糖尿病，它们是全球增长最快的流行病之一。对针对代谢性疾病的新分子实体（NMEs）的分析表明，新药的批准率在20世纪90年代中期有所上升，目前约为每年两种。这种增长主要归因于最近对先天性代谢缺陷治疗的重视。特别是，生物技术公司专注于罕见的遗传疾病，这些疾病通常用针对新途径且符合孤儿药地位的基于生物的NMEs进行治疗。相比之下，制药公司的NMEs开发倾向于针对非遗传疾病（如糖尿病）的传统小分子靶向治疗。
