COPENHAGEN, Denmark, August 6, 2026

Ascendis Pharma A/S announced encouraging updates across its achondroplasia development programs, highlighting positive Week 78 results from the Phase 2 COACH trial, durable findings from the pivotal ApproaCH study, progress in the reACHin infant trial, and continued commercial uptake of YUVIWEL® (navepegritide) in the United States. The company reported that once-weekly combination therapy with TransCon® CNP and TransCon hGH delivered sustained, unprecedented growth improvements while maintaining a favorable safety profile. The results reinforce the potential of the combination regimen as an important advancement for children with achondroplasia and support Ascendis Pharma’s long-term strategy to expand treatment options across multiple pediatric age groups. More than 170 unique U.S. patients had enrolled for YUVIWEL therapy by June 30, with over 65% receiving reimbursement approval, reflecting strong early commercial momentum.

Long-Term Clinical Data Strengthen TransCon CNP Development

The Phase 2 COACH trial demonstrated durable efficacy through Week 78, with children receiving TransCon CNP plus TransCon hGH achieving annualized growth velocity at or above the 97th percentile of children with average stature. Treatment-naïve patients achieved a 7.73 cm/year growth velocity, while previously treated patients maintained 7.67 cm/year, accompanied by sustained improvements in height Z-scores and body proportionality. Importantly, all 21 enrolled children completed 78 weeks of therapy and remained on treatment, underscoring encouraging tolerability. In parallel, Week 104 data from the pivotal ApproaCH trial confirmed durable improvements in height, lower-limb alignment, and body proportionality with once-weekly TransCon CNP monotherapy, while maintaining a safety profile comparable to placebo with no symptomatic hypotension, no treatment discontinuations, and only mild injection-site reactions. These findings continue to strengthen the clinical evidence supporting long-term treatment with TransCon CNP.

Regulatory Progress Expands Opportunities Across Age Groups

Ascendis Pharma also announced important development milestones beyond the ongoing studies. The company completed target enrollment for the pivotal reACHin trial, designed to support future regulatory submissions for infants aged 0 to under 2 years with achondroplasia, while the teACH study continues evaluating therapy in adolescents. Additionally, 96% of participants enrolled in the long-term AttaCH extension study remain on TransCon CNP treatment or have transitioned into the COACH combination trial, providing nearly six years of continuous clinical experience. In Europe, a regulatory decision on the Marketing Authorisation Application for TransCon CNP monotherapy is anticipated during the fourth quarter of 2026, representing another significant milestone in expanding global access to the therapy.

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YUVIWEL Uptake Supports Commercial Momentum

Alongside its clinical progress, Ascendis Pharma reported strong commercial adoption of YUVIWEL following its U.S. approval in February 2026. By the end of June, more than 170 unique patient enrollments had been generated by approximately 90 prescribing healthcare providers, with over 65% of patients approved for reimbursement, demonstrating encouraging payer acceptance. Company leadership emphasized that the combination of durable efficacy, favorable safety, convenient once-weekly dosing, and continued regulatory expansion positions TransCon CNP as a potentially transformative therapy for children with achondroplasia. With multiple late-stage clinical programs progressing simultaneously and long-term data continuing to mature, Ascendis Pharma is strengthening its leadership position in rare disease therapeutics while advancing toward broader global commercialization of its innovative TransCon technology platform.

Source:Ascendis Pharma, press release