
Clinical TrialJun 30, 2026, 08:32 AM
Ascendis Pharma TransCon CNP shows continued TFA improvements at Week 104
AI Summary
Ascendis Pharma announced new radiographic data from the pivotal ApproaCH Trial of once-weekly TransCon CNP in children with achondroplasia. The data, presented at ICCBH 2026, showed continued improvements in lower extremity alignment, specifically tibial-femoral angle (TFA), through up to two years of treatment (Week 104). Greater improvements were observed in children with preexisting genu varum, reinforcing the drug's potential to address serious complications of skeletal dysplasia. TransCon CNP was generally well tolerated, and the drug was approved by the US FDA in February 2026.
Key Highlights
- TransCon CNP showed continued improvements in tibial-femoral angle (TFA) through Week 104.
- Average TFA decreased from 9.1° at baseline to 6.9° at Week 104 in all treated children.
- Children with baseline TFA ≥ 5° saw average TFA decrease from 13.4° to 9.6° at Week 104.
- Mean absolute TFA change was -2.2 degrees over two years for all children.
- Mean absolute TFA change was -3.8 degrees over two years for children with baseline TFA ≥ 5°.
- Fibula-to-tibia length ratio remained stable, reflecting proportional growth of the lower leg.
- TransCon CNP was generally well tolerated with a low rate of ISRs and no symptomatic hypotension.
- US FDA approved TransCon CNP (YUVIWEL®) in February 2026 for achondroplasia.
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