Ascendis Pharma’s navepegritide receives a positive CHMP opinion for achondroplasia in children aged 2 and older, with a 1.49 cm/year growth velocity benefit versus placebo. EU approval remains pending.
Written By: Creola Gonsalves, MS, Biotech
Reviewed By: Pharmacally Editorial Team
Ascendis Pharma has received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), recommending marketing authorization for once-weekly TransCon CNP (navepegritide) in the European Union for children aged two years and older with achondroplasia whose growth plates remain open. The European Commission’s final decision on the marketing authorization application is expected in the fourth quarter of 2026.
If authorized, Ascendis expects to launch the treatment under the brand name YUVIWEL® in Germany during the first quarter of 2027. The recommendation marks a regulatory milestone for the company’s growth-disorder portfolio, although EU marketing authorization remains pending.
CHMP Recommendation and Supporting Evidence
The CHMP adopted its positive opinion after reviewing a marketing authorization application containing data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data.
The pivotal ApproaCH Phase 2b trial evaluated once-weekly subcutaneous navepegritide in 84 children aged two to 11 years with achondroplasia. Participants were randomized in a 2:1 ratio to receive navepegritide at 100 μg/kg/week or placebo during the 52-week double-blind period.
The primary endpoint was annualized growth velocity (AGV) at Week 52. Children receiving navepegritide had an AGV of 5.89 cm/year, compared with 4.41 cm/year in the placebo group. The least-squares mean treatment difference was 1.49 cm/year, with a 95% confidence interval of 1.05 to 1.93 cm/year and a statistically significant result (P<0.0001).
The trial also reported improvements in lower-limb alignment and body proportionality compared with placebo. These findings suggest that the treatment’s effects may extend beyond increasing linear growth, although the extent to which these changes translate into long-term functional benefits requires continued assessment.
Navepegritide demonstrated a safety and tolerability profile similar to placebo in the pivotal trial. This finding is reassuring, but the available evidence should be interpreted within the duration and population studied.
How Navepegritide Targets Achondroplasia
Achondroplasia is a genetic skeletal dysplasia associated with a gain-of-function variant in the fibroblast growth factor receptor 3 (FGFR3) gene. Excessive FGFR3 signalling impairs endochondral bone growth, contributing to disproportionate short stature and other skeletal abnormalities.
TransCon CNP is a prodrug of C-type natriuretic peptide (CNP), designed to provide sustained exposure to active CNP following once-weekly administration. CNP signalling counteracts the excessive FGFR3 pathway activity associated with achondroplasia, supporting the biological rationale for treatment.
The condition can also involve neurological, muscular and cardiorespiratory complications, including spinal abnormalities, sleep-disordered breathing, hearing problems and impaired physical function. These manifestations vary among individuals and across different stages of life.
Although the ApproaCH findings support an effect on growth and selected skeletal measures, they do not establish that navepegritide prevents or reverses all complications associated with achondroplasia.
Orphan Designation and European Launch Outlook
TransCon CNP received orphan medicine designation during development. Following the positive CHMP opinion, the EMA will review the available evidence to determine whether the designation can be maintained.
The European Commission will make the final decision on the marketing authorization application. Until that decision is issued, the CHMP recommendation should not be described as EU approval.
If authorization is granted, Ascendis expects Germany to be the first European market for YUVIWEL in the first quarter of 2027.
Clinical and Regulatory Significance
The positive opinion reflects a clinical evidence package that combines a statistically significant improvement in annualized growth velocity with supportive findings in body proportionality and lower-limb alignment. The once-weekly administration schedule also distinguishes the treatment approach, although dosing convenience alone does not establish comparative superiority over other therapies.
The remaining regulatory decision will determine whether navepegritide can enter the EU market. Longer-term evidence will remain important for understanding the durability of growth effects, functional outcomes and safety as children continue treatment.
References
Ascendis Pharma. Once-Weekly TransCon CNP (Navepegritide) Receives Positive CHMP Opinion for the Treatment of Achondroplasia in Children. October 16, 2026.
Savarirayan R, McDonnell C, Bacino CA, et al. Once-Weekly Navepegritide in Children with Achondroplasia: The ApproaCH Randomized Clinical Trial. JAMA Pediatrics. 2026;180(1):18–25. https://doi.org/10.1001/jamapediatrics.2025.4771
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared with Placebo in Children with Achondroplasia (ApproaCH), ClinicalTrials.gov ID NCT05598320
About the Writer
Creola Gonsalves (Linkedin) is an M.S. Biotechnology postgraduate with a strong interest in clinical research, evidence interpretation, and healthcare writing, with a focus on translating life-science knowledge into meaningful real-world insights.
She is trained in Good Clinical Practice (GCP), clinical research principles, and critical interpretation of randomized clinical trials, with certifications from NIH and Stanford University.
Her research background in biotechnological applications and microbial research strengthens her ability to understand scientific evidence and develop clear, accurate, and research-driven healthcare content.