Navepegritide for treating achondroplasia in children and adolescents aged 2 to 15 years old

Navepegritide is in clinical development for the treatment of achondroplasia. Achondroplasia is a genetic condition that causes impaired bone growth, particularly in the limbs and the spine. It is caused by a change in the fibroblast growth factor receptor 3 (FGFR3) gene which is a regulator of bone growth at cartilage growth plates (the areas from which bones grow in length). Reduction in bone growth causes clinical features associated with achondroplasia including short upper arms, leg bowing and short stature; this can result in long-term complications such as back and leg pain, apnoea (interruption of breathing, especially during sleep), obesity and curved spine. The FGRF3 gene is also expressed in tissues throughout the body, which can cause serious muscular, neurological, and cardiorespiratory complications. Currently there is no treatment for achondroplasia; symptoms can be managed through surgery to correct orthopaedic complications of achondroplasia such as leg bowing.