palovarotene

Palovarotene is a selective agonist of retinoic acid receptor gamma (RARγ), belonging to the class of retinoids. It is an orally administered small molecule developed for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues such as muscles, tendons, and ligaments are gradually replaced by bone. Palovarotene works by binding to RARγ in chondrogenic cells and chondrocytes, acting as a transcriptional repressor that decreases bone morphogenetic protein (BMP) signaling and inhibits the SMAD1/5/8 pathway. This mechanism reduces abnormal endochondral bone formation and allows for normal muscle tissue repair. The drug is approved for use in adults and children with FOP (females aged 8 years or older; males aged 10 years or older) to reduce new heterotopic ossification[1][3][4][5][6].

Brand names

Sohonos

Other names

palovaroteno