Ascendis Seeks EMA Approval For TransCon® CNP, A Potential Treatment For Children Living With Achondroplasia

Ascendis Pharma A/S has announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP (navepegritide), intended for the treatment of children with achondroplasia. Achondroplasia is a rare genetic disorder that leads to skeletal dysplasia and can cause significant challenges in growth, physical function, and overall quality of life. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) designed to provide continuous exposure of active CNP throughout the body, including the growth plates and skeletal muscle. Administered once weekly, it works by continuously inhibiting the overactive FGFR3 pathway, which plays a central role in achondroplasia.

Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma, stated, “The achondroplasia community has been clear about its need for early pharmacological interventions that improve the medical, functional, and quality of life complications. We look forward to engaging with the EMA during its review to bring TransCon CNP to patients in the European Union as soon as possible.”

The MAA submission is supported by results from three randomized, double-blind, placebo-controlled clinical studies and up to three years of open-label extension data. This includes findings from the pivotal ApproaCH Trial, which evaluated TransCon CNP in children with achondroplasia. Across these studies, the therapy was generally well tolerated, and no participants discontinued treatment due to the study drug. In parallel, TransCon CNP is also under priority review by the U.S. Food and Drug Administration (FDA) as a treatment for children with achondroplasia. The Prescription Drug User Fee Act (PDUFA) target date for the FDA’s decision is set for November 30, 2025.