Garetosmab for preventing abnormal bone formation in fibrodysplasia ossificans progressiva

Garetosmab is in clinical development for the prevention of abnormal bone formation, outside of the normal skeleton (heterotopic ossification), and soft-tissue flare-ups in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a very rare disease caused by a mutation in the gene ACVR1. The mutation results in the formation of unwanted bone in muscles, tendons, and ligaments throughout the body. Abnormal, misplaced and mis-shapen (heterotopic) bone can bridge across joints causing immobility, scoliosis, and other deformities. Patients usually require a wheelchair by the time they reach their 20s. Death often results in the 40s from complications, such as pneumonia, heart failure and loss of mobility in the chest, neck and jaw.
Garetosmab is administered intravenously. It is designed to attach to activin A, which stops activin A from interacting with ACVR1. This is expected to stop or reduce unwanted bone formation. If licensed, garetosmab will offer a treatment option for preventing abnormal bone formation and soft-tissue flare-ups in patients with FOP.