Ascendis Pharma has shared pivotal Week 52 results from its ApproaCH Trial evaluating once-weekly TransCon® CNP (navepegritide) in children with achondroplasia. The findings, now published in JAMA Pediatrics, highlight meaningful improvements in growth, limb alignment, and overall quality of life—without compromising safety.
What the Study Found?
The ApproaCH Trial was a randomized, double-blind, placebo-controlled study. It evaluated 84 children aged 2–11 years who received either TransCon CNP or placebo for 52 weeks.
Key Outcomes
- Higher annualized growth velocity (AGV): TransCon CNP significantly improved AGV compared to placebo.
- Better body proportionality: The treatment reduced the upper-to-lower body segment ratio from baseline to Week 52.
- Improved lower-limb alignment: Notable gains in tibial-femoral angle (TFA), mechanical axis deviation (MAD), and fibula-to-tibia length ratio.
- Improved daily functioning: Children on TransCon CNP reported numerical improvements across several ACEM quality-of-life domains.
- Safe and well tolerated: Most adverse events were mild or moderate. Injection site reactions were low. No symptomatic hypotension or bone fractures were reported.
Dr. Ravi Savarirayan noted that children receiving navepegritide “had significantly better growth and improvements in important health outcomes compared with placebo.” Community leaders echoed the importance of evaluating benefits beyond height.
Why This Matters for Families?
Families want to understand more than height outcomes. They want clarity on how a therapy affects the many medical challenges linked to achondroplasia.
Michael Hughes of Little People of America emphasized that including functional and quality-of-life endpoints “begins to fill that gap” and supports more informed healthcare decisions.
Ascendis Pharma’s Chief Medical Officer, Dr. Aimee Shu, reinforced this intent, saying the team aims to demonstrate benefits aligned with the priorities voiced by the achondroplasia community.
About TransCon CNP
TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide.
It is designed for once-weekly administration and provides continuous inhibition of the overactive FGFR3 pathway that drives achondroplasia. The long-acting profile aims to deliver CNP consistently to relevant tissues throughout the body.
The therapy is currently under Priority Review by the U.S. FDA with a target decision date of November 30, 2025, and is also being evaluated by the European Medicines Agency.
What Is Achondroplasia?
Achondroplasia is a rare genetic condition caused by an FGFR3 variant.
It affects more than 250,000 individuals worldwide and creates an imbalance between the FGFR3 and CNP pathways.
Although often labeled a bone growth disorder, achondroplasia affects multiple body systems.
Common medical challenges
- Spinal abnormalities
- Enlarged brain ventricles
- Muscle strength deficits
- Chronic ear infections and hearing issues
- Airway obstructions and sleep-disordered breathing
- Hip problems and leg bowing
- Chronic pain
- Cardiorespiratory complications
- High surgical burden across life stages
These complications impact quality of life, physical functioning, and psychosocial well-being.
About Ascendis Pharma
Ascendis Pharma is a global biopharmaceutical company using its TransCon technology to build best-in-class therapies for unmet medical needs. The company operates with a focus on Patients, Science, and Passion, and maintains facilities in Denmark, Europe, and the United States.
