TransCon CNP (navepegritide) - (ApproaCH)

June 30, 2026: Ascendis Pharma A/S (NASDAQ: ASND) reported new Week 104 radiographic results from the completed pivotal ApproaCH trial of once-weekly TransCon CNP (navepegritide) in children (ages 2–11; n=84) with achondroplasia, presented at the 12th International Conference on Children’s Bone Health (ICCBH 2026). In the randomized, double-blind, placebo-controlled 52-week period (TransCon CNP 100 µg/kg weekly vs placebo) followed by an open-label extension to Week 104, lower-extremity alignment improved through two years as measured by tibial-femoral angle (TFA) and TFA Z-scores, with greater improvements in children with baseline TFA ≥5° (preexisting genu varum). For children treated during the double-blind period, mean TFA decreased from 9.1° baseline to 7.7° (Week 52) and 6.9° (Week 104). In those switching from placebo at Week 52, mean TFA decreased to 10.1° by Week 104. Fibula-to-tibia length ratio remained stable, supporting proportional lower-leg growth. Safety through Week 104 was generally favorable (low, mild injection-site reactions; no symptomatic hypotension; no bone-age acceleration; no discontinuations).

The release notes FDA approval in February 2026 as YUVIWEL for increasing linear growth in pediatric achondroplasia (≥2 years, open epiphyses); the EMA MAA is under review with a decision anticipated in Q4 2026.