Palovarotene:A new drug for fibrodysplasia ossificans progressive

VernacularTitle:治疗进行性骨化性纤维发育不良的新药Palovarotene
Author: Ran WEI ¹ ; Chao-Yang CHEN ; Xuan-Ling ZHANG ; Ying ZHOU
Author Information

1. 北京大学第一医院药剂科,北京 100034
Keywords: Palovarotene; fibrodysplasia ossificans progressive; clinical research; safety
From: The Chinese Journal of Clinical Pharmacology 2024;40(5):745-748
CountryChina
Language:Chinese
Abstract: Progressive ossifying fibrous dysplasia(FOP)is a rare,hereditary,and progressive connective tissue disease characterized by heterotopic ossification(HO)formation in muscles,joints,tendons,and ligaments,leading to major joint stiffness,limited movement,deformity,and severe disability in patients.In August 2023,Palovarotene was approved by the US Food and Drug Administration(FDA)to reduce the formation of HO in adults and children aged 8 years and above for females and 10 years and above for males with FOP.It is currently the only approved curable drug worldwide.Palovarotene is a selective retinoic acid receptor γ(RAR-γ)agonist that reduces the formation of HO by inhibiting bone morphogenetic protein and SMAD 1/5/8 signaling.The pharmacological study,pharmacokinetics,clinical research,and safety are reviewed to comprehensively introduce Palovarotene.