COPENHAGEN, Denmark, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP (navepegritide) as a treatment for children with achondroplasia, a rare genetic condition that causes skeletal dysplasia and, for many affected individuals, significant health, physical functioning, and quality of life impacts. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.

The MAA is based on data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data, including results from the pivotal ApproaCH Trial of children with achondroplasia. In these trials, TransCon CNP has been generally well tolerated, with no discontinuations related to the study drug.

"The achondroplasia community has been clear about its need for early pharmacological interventions that improve the medical, functional, and quality of life complications," said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. "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."

In the United States, TransCon CNP as a treatment for children with achondroplasia is under priority review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target date of November 30, 2025.