The U.S. Food and Drug Administration (FDA) has extended the review period for Ascendis Pharma’s New Drug Application (NDA) for TransCon CNP (navepegritide), a therapy for children with Achondroplasia (dwarfism).
The agency notified the company that information submitted on November 5, 2025, related to the required post-marketing study, constitutes a “major amendment” to the NDA. As a result, the target action date under the Prescription Drug User Fee Act (PDUFA) has been postponed by three months to February 28, 2026.
According to Ascendis, the submission addressed all outstanding requests from the FDA, including a revised protocol for the post-marketing study, which had been the final item under discussion at the agency’s late-cycle meeting.
With this extension, stakeholders now await the final regulatory decision by February 28, 2026. If approved, TransCon CNP could become the first once-weekly therapy for children with achondroplasia in the United States, offering a potentially meaningful new treatment option.
