治疗进行性家族性肝内胆汁淤积症新药Bylvay

Bylvay, a new drug for the treatment of progressive familial intrahepatic cholestasis

odevixibat(商品名:Bylvay)是一种首创强效可逆性的回肠胆汁酸转运体(IBAT)抑制剂,具有特定的分子靶向性,能够减少回肠末端胆汁酸(主要是盐形式)的再吸收,可局部作用于肠道,能够在较低的剂量下产生作用,对全身影响较小。2021年7月被美国FDA批准上市,是全球首个用于治疗进行性家族性肝内胆汁淤积症(PFIC)的药物。现对其作用机制、药效学、药动学、临床研究及安全性做一介绍。

Odevixibat is a novel potent reversible ileal bile acid transporter(IBAT) inhibitor with specific molecular targeting property. It can reduce the reabsorption of bile acids at the end of ileum(mainly in the form of salt). Meanwhile, it can produce effect at a low dose and impact little on the whole body. It is the world’s first drug approved by the US FDA in July 2021 for the treatment of progressive familial intrahepatic cholestasis(PFIC). Its mechanism of action, pharmacodynamics, pharmacokinetics, clinical research and safety are introduced in this paper.