Ascendis shows combination therapy benefits in achondroplasia trial

Ascendis Pharma A/S (NASDAQ: ASND) reported data from its Phase 2 COACH trial showing that combining TransCon hGH with TransCon CNP produced improvements in arm span, spinal canal dimensions, and leg alignment in children with achondroplasia at 52 weeks.

The trial included 21 children aged 2 to 11 years, with 12 treatment-naïve patients and 9 previously treated with TransCon CNP monotherapy. For treatment-naïve children, arm span improved by 9.4 cm with combination therapy, while previously treated children showed 7.9 cm improvement.

Spinal canal measurements showed mean improvements in interpedicular distance of 1.7 mm for treatment-naïve children and 1.1 mm for previously treated children on combination therapy, compared to 0.6 mm for children on TransCon CNP alone in the ApproaCH trial.

The tibial femoral angle, which measures leg straightening, improved by an average of 3.0 degrees in treatment-naïve children receiving combination therapy versus 1.3 degrees for those on monotherapy in the ApproaCH trial. Children who had received long-term TransCon CNP monotherapy maintained normal tibial femoral angle ranges.

TransCon CNP received FDA approval in February 2026 under the trade name YUVIWEL for achondroplasia treatment and is under European review. TransCon hGH is approved as SKYTROFA for growth hormone deficiency but remains investigational for achondroplasia.

The company previously reported that the combination therapy achieved mean annualized growth velocity exceeding the 97th percentile of average stature children without compromising safety or accelerating bone age. All 21 children completed 52 weeks of treatment and continue on therapy.

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