공시 • May 09
Ascendis Pharma A/S Announces New 2-Year Data from Pivotal ApproaCH Trial of TransCon® CNP (Navepegritide) Show Pronounced Gains in Growth Outcomes in Children with Achondroplasia Aged =5 Years
Ascendis Pharma A/S announced new data from a subgroup analysis showing that children with achondroplasia =5 years of age at enrollment treated with once-weekly TransCon CNP (navepegritide) in its pivotal ApproaCH Trial demonstrated significantly greater annualized growth velocity (AGV) compared to placebo at Week 52, and sustained these growth improvements through up to two years of treatment. The safety profile for this subgroup through up to two years of treatment was similar to the overall population, with a low rate of injection site reactions (ISRs, all mild), no symptomatic hypotension, and no acceleration of bone age. The data follow previously reported Week 104 results showing consistent improvements in growth and body proportionality in the overall population, and expand on data recently presented by M. Jennifer Abuzzahab, M.D. during PES 2026, the annual meeting of the Pediatric Endocrine Society. ApproaCH was a randomized, double-blind, placebo-controlled pivotal trial in 84 children with achondroplasia aged 2–11 years, investigating TransCon CNP (100 µg/kg once-weekly) versus placebo for 52 weeks, followed by a 52-week open-label extension (OLE) period in which all participants received TransCon CNP through Week 104. Fifty-three of the 84 children were =5 years of age at the time of their enrollment in the trial. Subgroup of children aged =5 years at enrollment: AGV (cm/year) LS Mean Week 52: TransCon CNP(n=36) 5.79, Placebo/TransCon CNP1 (n=17) 4.02; Observed Mean3 Week 52: TransCon CNP(n=36) 5.84, Placebo/TransCon CNP1 (n=17) 3.88; Observed Mean3 Week 104: TransCon CNP(n=36) 5.71, Placebo/TransCon CNP1 (n=17) 5.53; TransCon CNP vs. Placebo, Treatment Difference +1.78 [95% CI] [1.22, 2.33]; +1.972 [95% CI] [1.37, 2.56]. ACH-Specific Height Z-score, Change from Baseline: LS Mean Week 52: TransCon CNP (n=36) +0.38, Placebo/TransCon CNP1 (n=17) +0.07; Observed Mean3 Week 52: TransCon CNP (n=36) +0.38, Placebo/TransCon CNP1 (n=17) +0.07; Observed Mean3 Week 104: TransCon CNP (n=36) +0.75, Placebo/TransCon CNP1 (n=17) +0.46. CDC-Based Height Z-score, Change from Baseline: LS Mean Week 52: TransCon CNP (n=36) +0.28, Placebo/TransCon CNP1 (n=17) -0.05; Observed Mean3 Week 52: TransCon CNP (n=36) +0.26, Placebo/TransCon CNP1 (n=17) -0.02; Observed Mean3 Week 104: TransCon CNP (n=36) +0.58, Placebo/TransCon CNP1 (n=17) +0.36. TransCon CNP vs. Placebo, Treatment Difference2: ACH-Specific Height Z-score, Change from Baseline: +0.31 [95% CI] [0.20, 0.42]; +0.30 [95% CI] [0.18, 0.42]. CDC-Based Height Z-score, Change from Baseline: +0.32 [95% CI] [0.20, 0.44]; +0.29 [95% CI] [0.14, 0.44]. Through up to two years of treatment, the safety profile in children =5 years was similar to the overall population, with a low rate of ISRs (all mild), no symptomatic hypotension, and no acceleration of bone age. Most adverse events in TransCon CNP-treated children were mild or moderate, with none leading to treatment discontinuation or withdrawal from the trial. TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. In February 2026, TransCon CNP was approved by the U.S. Food & Drug Administration (FDA) under the trade name YUVIWEL to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL is under review by the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026.