BPS-804 is a human monoclonal antibody targeting sclerostin, which is being developed to improve bone quality and thereby reduce fractures in the orphan disease osteogenesis imperfecta (OI).
OI is a rare genetic disorder, commonly known as brittle bone disease, which is characterized by fragile bones that break easily. In addition to fractures, individuals with OI often have muscle weakness, hearing loss, fatigue, joint laxity, curved bones, scoliosis, and short stature. The majority of cases of OI (up to 90 %) are caused by a dominant mutation in the genes coding for type I collagen, a key component of healthy bone. Current treatment of OI is based on supportive care, focusing on minimizing fractures and maximizing mobility. To date, there are no approved treatments that address the underlying cause of bone fragility.
- Based on Osteogenesis Imperfecta Foundation estimates
- Based on Orphanet estimates
- Shapiro J (2014) Osteogenesis Imperfecta: A Translational Approach to Brittle Bone Disease. Academic Press. Chapter 2: p15-22