Ascendis Pharma Hit By FDA Delay As TransCon CNP Decision Slips To 2026

Core Viewpoint - The FDA has extended the review period for Ascendis Pharma's New Drug Application for TransCon CNP, a treatment for children with achondroplasia, by three months to February 28, 2026, due to a major amendment related to post-marketing requirements [1][3]. Company Overview - Ascendis Pharma is focused on developing treatments for rare diseases, with TransCon CNP being a key product aimed at addressing achondroplasia, the most common form of skeletal dysplasia caused by a mutation in the FGFR3 gene [2][4]. - The company has reported positive results from its ApproaCH Trial, demonstrating significant improvements in annualized growth velocity and health-related quality of life for children treated with TransCon CNP compared to placebo [4][5]. Regulatory Update - The FDA's extension of the PDUFA target action date is a response to the major amendment submitted by Ascendis Pharma, which included a revised protocol for the post-marketing study [3]. - The company has addressed all outstanding requests from the FDA, indicating proactive engagement in the regulatory process [3]. Clinical Data - The pivotal week 52 results from the ApproaCH Trial were published in JAMA Pediatrics, highlighting the efficacy of TransCon CNP in improving growth metrics and overall health outcomes for children with achondroplasia [4][5]. - The treatment showed a safety and tolerability profile similar to that of placebo, reinforcing its potential as a viable therapeutic option [5]. Market Performance - Ascendis Pharma's stock price increased by 1.44%, reaching $213.03 at the time of publication, reflecting positive market sentiment regarding the company's developments [7].