Ascendis Pharma A/S (NASDAQ: ASND) announced results from a subgroup analysis of its pivotal ApproaCH trial showing that children aged 5 years and older with achondroplasia treated with TransCon CNP demonstrated sustained growth improvements through two years of treatment.

The analysis included 53 children aged 5 years and older from the 84-participant trial. Children receiving TransCon CNP showed an annualized growth velocity of 5.79 cm/year at week 52 compared to 4.02 cm/year for placebo, representing a treatment difference of 1.78 cm/year. Growth velocity remained consistent through week 104 at 5.71 cm/year.

The trial was a randomized, double-blind, placebo-controlled study investigating TransCon CNP at 100 µg/kg once weekly versus placebo for 52 weeks, followed by a 52-week open-label extension where all participants received the treatment.

Height improvements were measured using both achondroplasia-specific and CDC-based height Z-scores. At week 52, TransCon CNP showed a treatment difference of +0.31 for achondroplasia-specific height Z-score change from baseline and +0.32 for CDC-based measurements, both with statistical significance.

The safety profile for this subgroup was consistent with the overall population through two years, with mild injection site reactions, no symptomatic hypotension, and no acceleration of bone age. Most adverse events were mild or moderate, with none leading to treatment discontinuation.

TransCon CNP received FDA approval in February 2026 under the trade name YUVIWEL for increasing linear growth in pediatric patients 2 years and older with achondroplasia. The European Medicines Agency is reviewing the Marketing Authorisation Application with a decision expected in the fourth quarter of 2026.