FDA Delays Decision on Ascendis’ Dwarfism Drug by Three Months
The FDA has delayed its decision on Ascendis Pharma's dwarfism drug candidate TransCon CNP (navepegritide) by three months, moving the decision deadline from November 30, 2025, to February 28, 2026.
The delay was triggered by Ascendis submitting new information on November 5, 2025, related to post-marketing requirements, which the FDA classified as a major amendment to the New Drug Application.
The only outstanding item discussed with the FDA was a request for a revised protocol for the post-marketing study.
Analysts believe the delay is procedural and not due to safety or efficacy concerns, and expect approval is still likely.
If approved, TransCon CNP will compete with BioMarin's Voxzogo as a treatment for children with achondroplasia.
TransCon CNP is a once-weekly prodrug of C-type natriuretic peptide (CNP), designed to stimulate bone growth in children with achondroplasia.