New Data Shows Improvements in Growth and Bone Morphometry in Children with Achondroplasia Treated with TransCon® CNP (Navepegritide)

Core Insights - Ascendis Pharma announced new data from the ApproaCH Trial showing improvements in growth and bone morphometry in children with achondroplasia, highlighting the efficacy of TransCon CNP (navepegritide) [1][3][4] Group 1: Trial Details - The ApproaCH Trial was a double-blind placebo-controlled study involving 84 children aged 2-11 years, randomized in a 2:1 ratio for 52 weeks, followed by an open-label extension period [3] - At Week 52, TransCon CNP demonstrated superiority over placebo in annualized growth velocity (AGV) with a safety profile comparable to placebo, including low rates of injection site reactions and no serious adverse events [3] Group 2: Bone Morphometry Improvements - TransCon CNP showed significant improvements in bone morphometry at Week 52, including better lower limb alignment, proportional growth, and increased spinal canal dimensions compared to placebo [4] Group 3: Company Vision and Future Goals - The company aims to deliver benefits beyond linear growth for children with achondroplasia and is committed to understanding how altering the trajectory of skeletal dysplasia could reduce future complications associated with the condition [5] - Ascendis Pharma focuses on applying its innovative TransCon technology platform to develop new therapies that address unmet medical needs, with a commitment to patient care and scientific advancement [7]