Announcement • Jun 30

Ascendis Pharma A/S Announces New Radiographic Data from Week 104 of the Completed Pivotal Approach Trial of Once-Weekly Transcon Cnp in Children with Achondroplasia

Ascendis Pharma A/S announced new radiographic data from Week 104 from the completed pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia. In the trial, TransCon CNP-treated children demonstrated continued improvements in lower extremity alignment through up to two years of treatment, including improvements in tibial-femoral angle (TFA). As previously reported, improvements in annualized growth velocity were maintained and ACH-specific height Z-score increased with TransCon CNP treatment through Week 104. 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 an open-label extension (OLE) in which all participants received TransCon CNP through Week 104. Radiographic assessments of lower extremity alignment were conducted at baseline, Week 52, and Week 104. Improvements in TFA and TFA Z-scores continued through Week 104 and were greater in children with baseline TFA = 5°. The average TFA in all children treated with TransCon CNP during the double-blind period was 9.1° at baseline, decreasing to 7.7° at Week 52 and 6.9° at Week 104, reflecting a mean absolute change of -2.2 degrees over the two-year treatment period. In the subgroup of children with baseline TFA = 5° (reflecting children with preexisting genu varum), the average TFA was 13.4° at baseline, decreasing to 11.3° at Week 52 and 9.6° at Week 104, reflecting a mean absolute change of -3.8 degrees over the two-year treatment period. The average TFA in children switching from placebo to TransCon CNP treatment at Week 52 was 11.5° at baseline, increasing to 11.8° at Week 52 and decreasing with TransCon CNP treatment to 10.1° at Week 104, reflecting a mean absolute change of -1.7 degrees during the OLE period. In the subgroup of children with baseline TFA = 5°, the average TFA was 18.2° at baseline, increasing to 18.7° at Week 52 and decreasing with treatment to 14.9° at Week 104, reflecting a mean absolute change of -3.8 degrees during the OLE period. In children treated with TransCon CNP in the double-blind period, mean TFA Z-score was 3.62 at baseline and decreased to 3.15 at Week 52 and 2.96 at Week 104, reflecting a mean absolute change of -0.66 over the two-year treatment period. In the subgroup of children with baseline TFA = 5°, mean TFA Z-score was 5.40 at baseline and decreased to 4.67 at Week 52 and 4.36 at Week 104, reflecting a mean absolute change of -1.04 over the two-year treatment period. In children switching from placebo to TransCon CNP at Week 52, mean TFA Z-score was 4.08 at baseline, increased to 4.76 at Week 52, and decreased to 4.28 at Week 104, reflecting a mean absolute change of -0.48 from Week 52 to Week 104. In the subgroup of children with baseline TFA = 5°, mean TFA Z-score was 6.13 at baseline, increased to 7.56 at Week 52, and decreased to 6.35 at Week 104, reflecting a mean absolute change of -1.21 from Week 52 to Week 104. Results also showed that fibula-to-tibia length ratio remained stable in the overall clinical trial population during the open-label extension, reflecting proportional growth of the lower leg. Through up to two years of treatment, TransCon CNP was generally well tolerated, 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 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.

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