Ascendis Submits Marketing Authorisation Application to the European Medicines Agency for TransCon® CNP for Treatment of Children with Achondroplasia

Core Insights - Ascendis Pharma has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP as a treatment for children with achondroplasia, a rare genetic condition impacting skeletal development and quality of life [1][2] - TransCon CNP is designed to provide continuous inhibition of the overactive FGFR3 pathway, administered once weekly to enhance the treatment's effectiveness [1][2] - The MAA is supported by data from three randomized clinical trials, including the pivotal ApproaCH Trial, demonstrating that TransCon CNP has been generally well tolerated [2] Company Overview - Ascendis Pharma is a global biopharmaceutical company focused on innovative therapies using its TransCon technology platform, aiming to address unmet medical needs [5] - The company is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States [5] Industry Context - Achondroplasia affects over 250,000 individuals worldwide and is associated with various medical complications beyond skeletal dysplasia, including muscular, neurological, and cardiorespiratory issues [4] - The need for early pharmacological interventions in the achondroplasia community has been emphasized, highlighting the importance of treatments like TransCon CNP [3]