Fidrisertib

Fidrisertib (IPN60130) is an investigational oral small molecule drug developed for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare and severely disabling genetic disorder characterized by heterotopic ossification—the formation of bone in soft tissues where bone should not exist. FOP is caused by mutations in the ACVR1 gene, leading to excessive signaling through the bone morphogenetic protein (BMP) pathway. Fidrisertib acts as a selective inhibitor of activin receptor-like kinase 2 (ALK2/ACVR1), specifically targeting mutant forms responsible for disease pathology. By inhibiting this receptor, fidrisertib aims to reduce abnormal BMP signaling and prevent new heterotopic bone formation. The drug is currently being evaluated in phase 2 clinical trials for both adult and pediatric patients with FOP[1][2][3][5].

Other names

IPN60130IPN-60130IPN 60130BLU-782BLU782BLU 782