Ascendis Pharma Achondroplasia Programs Show Strong Clinical Results
Ascendis Pharma provided positive updates on its achondroplasia programs, including durable growth and improved body proportionality from the combination therapy (TransCon CNP + TransCon hGH) in the COACH trial, with 100% patient retention at Week 78. The TransCon CNP monotherapy also demonstrated durable height improvements and benefits beyond height in the ApproaCH trial over 104 weeks, with a favorable safety profile. The company also reported progress on the US launch of YUVIWEL (navepegritide), with over 170 patient enrollments and 65% reimbursement approval, and anticipates an EU regulatory decision for TransCon CNP monotherapy in Q4 2026.
Key Highlights
- Combination therapy showed mean AGV of 7.73 cm/year for treatment-naïve cohort at Week 78.
- Combination therapy showed mean AGV of 7.67 cm/year for CNP-experienced cohort at Week 78.
- TransCon CNP monotherapy demonstrated durable height improvements over 104 weeks.
- TransCon CNP monotherapy had 0.35 injection-site reactions per person-year.
- Completed target enrollment for pivotal reACHin Trial for infants 0 to <2 years.
- EU regulatory decision for TransCon CNP monotherapy anticipated in Q4 2026.
- YUVIWEL US launch has over 170 patient enrollments with 65%+ reimbursement approval.
- 100% of 21 children completed 78 weeks of combination therapy in COACH trial.
Price Impact
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