COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has submitted its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle.
The filing 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.
“We are pleased to share clinical data with the FDA demonstrating that, in addition to increased growth velocity, treatment with TransCon CNP was associated with reduced health-related burden, stronger muscle function, and straightening of abnormal leg bowing for the majority of treated children,” said Aimee Shu, M.D., Executive Vice President and Chief Medical Officer at Ascendis Pharma. “In addition to once-weekly administration, these outcomes and a safety and tolerability profile comparable to placebo support TransCon CNP’s potential to be recognized as a best-in-class treatment for achondroplasia.”
Ascendis is on track to submit its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during Q3 2025.
About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal deformities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
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