Ascendis Pharma A/S released 52-week data from its ApproaCH trial, showing that TransCon CNP (navepegritide) improved growth and bone morphometry in children with achondroplasia. The investigational drug, a once-weekly prodrug of C-type natriuretic peptide (CNP), demonstrated superior annualized growth velocity compared to placebo, with a comparable safety profile. Positive results included improvements in lower limb alignment, proportional growth, and spinal canal dimensions.
This data is crucial for the achondroplasia community because the condition affects more than 250,000 people globally and current treatments are limited. Achondroplasia, stemming from a fibroblast growth factor receptor 3 (FGFR3) variant, causes not just bone growth issues but also muscular, neurological, and cardiorespiratory complications requiring multiple surgeries and procedures throughout life. TransCon CNP’s potential to improve bone morphometry and address broader skeletal dysplasia could significantly reduce these complications and improve the quality of life for those affected.
The ApproaCH trial, a double-blind placebo-controlled study, enrolled 84 children aged 2–11. Key findings at Week 52 showed improvements in annualized growth velocity and various aspects of bone morphometry. Importantly, the safety profile was comparable to placebo, with low rates of injection site reactions, no treatment-related serious adverse events, and no adverse effects on blood pressure, bone fractures, or bone age acceleration. The open-label extension period of the trial will provide further data on the long-term efficacy and safety of TransCon CNP.
These positive results pave the way for potential regulatory submission and eventual commercialization of TransCon CNP. This therapy offers a promising new treatment option for children with achondroplasia, addressing not just height, but potentially a broader spectrum of skeletal complications. Further research will focus on understanding the long-term effects of TransCon CNP on overall health and functional outcomes in this population.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
