Ascendis Pharma A/S 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 affecting more than 250,000 individuals worldwide. The investigational prodrug of C-type natriuretic peptide (CNP) is administered once weekly and designed for continuous inhibition of the overactive FGFR3 pathway in achondroplasia.
Clinical Trial Foundation
The MAA submission is supported by comprehensive clinical data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. The evidence package includes results from the pivotal ApproaCH Trial of children with achondroplasia. Throughout these trials, TransCon CNP has been generally well tolerated, with no discontinuations related to the study drug.
TransCon CNP works by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. This mechanism targets the underlying pathophysiology of achondroplasia by addressing the imbalance in FGFR3 and CNP signaling pathways.
Addressing Unmet Medical Need
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that causes skeletal dysplasia and significant health complications. While historically considered a bone growth disorder, the FGFR3 variant is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia.
Medical complications vary across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, 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 complications persist or worsen in adulthood, having 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.
Regulatory Progress
"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.
TransCon Technology Platform
Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransCon technology platform to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. The company is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States.
