Ascendis Announces Longer FDA Review Timeline For TransCon CNP (navepegritide) For Pediatric Achondroplasia

Ascendis Pharma A/S, announced that the U.S. Food & Drug Administration (FDA) has informed the company that the information it submitted on November 5, 2025, regarding a post-marketing requirement for TransCon CNP (navepegritide), has been classified as a major amendment to the New Drug Application (NDA). TransCon CNP is currently under FDA review as a potential treatment for children with achondroplasia. As a result of this major amendment, the FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date by three months, setting the new date for February 28, 2026.

According to Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma, the company has now responded to all outstanding FDA requests. This includes submitting a revised protocol for the required post-marketing study, which was the only item the agency brought forward during the late-cycle meeting. He emphasized that Ascendis Pharma remains committed to working closely with the FDA to complete the remaining elements of the post-marketing requirement. The company aims to bring this innovative therapy to children in the United States as efficiently as possible once the review process is finalized.