COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today 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.
“We are pleased to see confirmation of the expected sustained growth improvements in these children, with a consistent safety and tolerability profile,” said Aimee D. Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. “These results, along with the improved skeletal alignment and body proportionality and positive changes in health-related quality of life previously reported for the overall populationi further highlight TransCon CNP’s ability to promote healthy, proportional growth in children with achondroplasia across age groups.”
ApproaCH Trial Design
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.
Highlights of the ApproaCH Trial Data Through Week 104
Subgroup of children aged ≥5 years at enrollment
| AGV (cm/year) | |||
| LS Mean | Observed Mean3 | ||
| Week 52 | Week 52 | Week 104 | |
| TransCon CNP(n=36) | 5.79 | 5.84 | 5.71 |
| Placebo/TransCon CNP1 (n=17) | 4.02 | 3.88 | 5.53 |
| TransCon CNP vs. Placebo, Treatment Difference [95% CI] |
+1.78 [1.22, 2.33] p<0.0001 |
+1.972 [1.37, 2.56] p<0.0001 |
– |
| ACH-Specific Height Z-score, Change from Baseline |
CDC-Based Height Z-score, Change from Baseline |
|||||
| LS Mean | Observed Mean3 | LS Mean | Observed Mean3 | |||
| Week 52 | Week 52 | Week 104 | Week 52 | Week 52 | Week 104 | |
| TransCon CNP (n=36) | +0.38 | +0.38 | +0.75 | +0.28 | +0.26 | +0.58 |
| Placebo/TransCon CNP1 (n=17) | +0.07 | +0.07 | +0.46 | -0.05 | -0.02 | +0.36 |
| TransCon CNP vs. Placebo, Treatment Difference2 [95% CI] |
+0.31 [0.20, 0.42] p<0.0001 |
+0.30 [0.18, 0.42] p<0.0001 |
– | +0.32 [0.20, 0.44] p<0.0001 |
+0.29 [0.14, 0.44] p=0.0004 |
– |
Note: The observed mean is a simple average of recorded measurements; the LS mean is a model-based estimated average that adjusts for selected variables, typically baseline patient characteristics, enabling a more balanced comparison across arms of a clinical trial.
1 Week 104 data reflects placebo patients that crossed over to TransCon CNP treatment at Week 52
2 Not presented at PES 2026; included for context
3 Treatment differences between TransCon CNP and placebo were estimated from a T-test
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.
A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com.
About TransCon CNP
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.
About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, and is associated with an increased risk of muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia can vary from individual to individual and across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and reduced stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; some of which persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns.
About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.
Investor Contacts
Chad Fugere – Ascendis Pharma
Media Contact
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