Wedoany.com Report-Nov. 27, The U.S. Food and Drug Administration has extended its review of Ascendis Pharma's TransCon CNP, a treatment for children with achondroplasia, the most common form of dwarfism. The new decision deadline is February 28, 2026, representing a three-month extension from the original target.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020

Ascendis announced the delay on Tuesday, explaining that the FDA classified additional information submitted on November 5 as a major amendment. The submission included a revised plan for a required post-approval follow-up study.

The company stated that it has addressed all outstanding questions from the agency and will continue close collaboration to complete the remaining requirements.

CEO Jan Mikkelsen said Ascendis had answered all remaining questions from the agency, including providing a revised plan for the post-approval study, and would continue to work closely with the FDA to finalize the requirements.

Achondroplasia is a rare genetic condition caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, which disrupts normal bone growth and leads to short stature and other skeletal complications.

TransCon CNP is designed to deliver continuous exposure to C-type natriuretic peptide (CNP), a naturally occurring molecule that counteracts the growth-inhibiting effects of the FGFR3 mutation and promotes bone development.

The therapy is administered once weekly and aims to improve linear growth and overall health outcomes in children with achondroplasia. The extension allows the FDA additional time to review the updated study plan while maintaining the ongoing evaluation of safety and efficacy data from previous clinical trials.

Ascendis remains engaged with the agency to support a thorough and timely completion of the review process.