Globenewswire·2025-11-17 21:01Core Insights - Ascendis Pharma announced pivotal Week 52 results from the ApproaCH Trial of TransCon® CNP (navepegritide) in children with achondroplasia, showing significant improvements in annualized growth velocity compared to placebo [1][4][5] - The trial demonstrated favorable impacts on body proportionality and health-related quality of life, with a safety profile similar to placebo [1][5][6] Group 1: Trial Results - The ApproaCH Trial was a randomized, double-blind, placebo-controlled study involving 84 children aged 2-11, with a 2:1 randomization to receive either TransCon CNP at 100 μg/kg/week or placebo over 52 weeks [3] - Treatment with TransCon CNP resulted in a significant increase in annualized growth velocity (AGV) and improvements in lower-limb alignment and body proportionality compared to placebo [1][4] - The trial also reported improvements in health-related quality of life across several domains, without accelerating bone age or negatively affecting spinal curvature [5] Group 2: Treatment Profile - TransCon CNP is an investigational prodrug designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia, administered once weekly [2][6] - The treatment showed a low rate of injection site reactions and no observed symptomatic hypotension or bone fractures during the trial [5] - The FDA has placed TransCon CNP under Priority Review, with a target date of November 30, 2025, and it is also under review by the European Medicines Agency [6] Group 3: Condition Overview - Achondroplasia is a rare genetic condition affecting over 250,000 individuals globally, leading to various medical complications beyond skeletal dysplasia [7][8] - Complications include spinal abnormalities, impaired muscle strength, and chronic pain, which can significantly impact quality of life and require multiple surgeries [8]