Ascendis Submits U.S. NDA for TransCon CNP (Navepegritide) for the Treatment of Children with Achondroplasia

Core Insights - Ascendis Pharma has submitted a New Drug Application (NDA) to the FDA for TransCon CNP, aimed at treating children with achondroplasia, supported by data from three clinical trials and up to three years of open-label extension data [1][2] - The company plans to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in Q3 2025 [3] Group 1: Clinical Data and Benefits - Clinical data indicates that TransCon CNP not only increases growth velocity but also reduces health-related burdens, enhances muscle function, and corrects leg bowing in most treated children [3] - The treatment is administered once weekly and has a safety and tolerability profile comparable to placebo, positioning it as a potential best-in-class treatment for achondroplasia [3] Group 2: Achondroplasia Overview - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to various complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - Complications can include spinal deformities, impaired muscle strength, and chronic pain, significantly impacting quality of life and requiring multiple surgeries [4] Group 3: Company Background - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5]