COPENHAGEN, Denmark — May 6, 2026 — Leads & Copy —
Ascendis Pharma A/S (Nasdaq: ASND) announced new data from a subgroup analysis of its pivotal ApproaCH Trial, revealing that children with achondroplasia aged 5 years and older at enrollment, who were treated with once-weekly TransCon CNP (navepegritide), demonstrated significantly greater annualized growth velocity (AGV) compared to those on placebo at Week 52. These growth improvements were sustained for up to two years of treatment.
The subgroup’s safety profile remained consistent with the overall trial population, showing a low rate of mild injection site reactions (ISRs), no symptomatic hypotension, and no acceleration of bone age over the two-year period. These findings build upon previously reported Week 104 results, which indicated consistent growth and body proportionality improvements in the broader study group. The data were recently presented by M. Jennifer Abuzzahab, M.D., at the Pediatric Endocrine Society (PES) 2026 annual meeting.
Aimee D. Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma, expressed satisfaction with the confirmation of sustained growth improvements and the consistent safety and tolerability profile observed in the children. She noted that these results, along with improved skeletal alignment, body proportionality, and positive changes in health-related quality of life previously reported, further emphasize TransCon CNP’s potential to promote healthy, proportional growth in children with achondroplasia across different age groups.
The ApproaCH trial was a randomized, double-blind, placebo-controlled study involving 84 children aged 2–11 years with achondroplasia. Participants were administered TransCon CNP (100 µg/kg once-weekly) or placebo for 52 weeks, followed by a 52-week open-label extension (OLE) where all participants received TransCon CNP up to Week 104. Of the participants, 53 were at least 5 years old at enrollment.
Key highlights from the ApproaCH trial data through Week 104 for the subgroup of children aged ≥5 years at enrollment include:
At Week 52, the TransCon CNP group (n=36) showed an LS Mean AGV of 5.79 cm/year and an observed mean of 5.84 cm/year, which was sustained at Week 104 with an observed mean of 5.71 cm/year. In comparison, the placebo/TransCon CNP group (n=17) had an LS Mean AGV of 4.02 cm/year and an observed mean of 3.88 cm/year at Week 52, increasing to an observed mean of 5.53 cm/year at Week 104 after crossing over to TransCon CNP treatment.
The treatment difference between TransCon CNP and placebo was +1.78 [95% CI: 1.22, 2.33] (p<0.0001) at Week 52 and +1.97 [1.37, 2.56] (p<0.0001) at Week 104.
For the ACH-Specific Height Z-score, the TransCon CNP group showed a change from baseline of +0.38 (LS Mean and observed mean) at Week 52, increasing to +0.75 (LS Mean) at Week 104. The placebo/TransCon CNP group had a change of +0.07 (LS Mean and observed mean) at Week 52, which increased to +0.46 (LS Mean) at Week 104.
The treatment difference between TransCon CNP and placebo was +0.31 [0.20, 0.42] (p<0.0001) at Week 52 and +0.30 [0.18, 0.42] (p<0.0001) at Week 104.
The CDC-Based Height Z-score also showed improvements in the TransCon CNP group, with a change from baseline of +0.28 (LS Mean) and +0.26 (observed mean) at Week 52, increasing to +0.58 (LS Mean) at Week 104. The placebo/TransCon CNP group showed a change of -0.05 (LS Mean) and -0.02 (observed mean) at Week 52, increasing to +0.36 (LS Mean) at Week 104.
The treatment difference between TransCon CNP and placebo was +0.32 [0.20, 0.44] (p<0.0001) at Week 52 and +0.29 [0.14, 0.44] (p=0.0004) at Week 104.
Ascendis Pharma’s TransCon CNP, now FDA-approved as YUVIWEL®, is designed to provide continuous exposure of active CNP to counteract overactive FGFR3 signaling in achondroplasia. The European Medicines Agency is reviewing Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL, with a decision expected in the fourth quarter of 2026.
Achondroplasia is a rare genetic condition affecting over 250,000 people worldwide, stemming from a fibroblast growth factor receptor 3 (FGFR3) variant. This variant leads to muscular, neurological, and cardiorespiratory complications, alongside skeletal dysplasia. The condition can lead to spinal abnormalities, impaired muscle strength, hearing deficits, upper airway obstructions, and chronic pain.
Ascendis Pharma is a global biopharmaceutical company employing its TransCon technology platform to address unmet medical needs. Headquartered in Copenhagen, Denmark, Ascendis Pharma has additional facilities in Europe and the United States.
Source: Ascendis Pharma
