Ascendis Pharma A/S announced new data from Week 52 of its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia. The data, presented at the ESPE & ESE 2025 joint congress, demonstrated significant improvements in growth and bone morphometry.
In the double-blind placebo-controlled trial, TransCon CNP showed superiority over placebo in annualized growth velocity (AGV). The treatment also led to improvements in lower limb alignment, proportional growth, and increases in spinal canal dimensions, indicating benefits beyond linear growth.
The safety and tolerability profile of TransCon CNP was comparable to placebo, with a low rate of injection site reactions, no treatment-related serious adverse events, no symptomatic hypotension, and no acceleration of bone age versus chronological age. These results further support the potential of TransCon CNP as a transformative treatment for achondroplasia.
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