Clinical TrialJun 30, 2026, 04:11 PM
Ascendis Pharma TransCon CNP shows continued achondroplasia improvements
AI Summary
Ascendis Pharma A/S announced new radiographic data from Week 104 of the pivotal ApproaCH Trial for once-weekly TransCon CNP (navepegritide) in children with achondroplasia. The data demonstrated continued improvements in lower extremity alignment, including tibial-femoral angle (TFA), through up to two years of treatment. Improvements in annualized growth velocity and ACH-specific height Z-score were also maintained. TransCon CNP was generally well tolerated, with a low rate of mild injection site reactions and no symptomatic hypotension or acceleration of bone age.
Key Highlights
- TransCon CNP showed continued improvements in lower extremity alignment through Week 104.
- Average tibial-femoral angle (TFA) decreased by 2.2 degrees over two years in all treated children.
- Children with baseline TFA ≥ 5° saw an average TFA decrease of 3.8 degrees over two years.
- Patients switching from placebo to TransCon CNP showed a 1.7-degree TFA decrease during the OLE period.
- Mean TFA Z-score decreased by 0.66 over two years in the TransCon CNP treated group.
- Fibula-to-tibia length ratio remained stable, indicating proportional lower leg growth.
- TransCon CNP was generally well tolerated with low rates of mild injection site reactions.
- No symptomatic hypotension or acceleration of bone age was observed with TransCon CNP.
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