ASND News & Events

Ascendis Pharma announced positive Week 52 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. investigating the safety, tolerability, and efficacy of once-weekly TransCon hGH vs. daily somatropin in prepubertal children with Turner syndrome. Turner syndrome is the most common congenital sex chromosomal condition in females, with an estimated prevalence of 1 out of every 2,000 to 2,500 live female births. At Week 52, children treated with TransCon hGH demonstrated improved annualized height velocity similar to daily somatropin, independent of starting dose, with an LS mean AHV of 9.05 cm/year for all TransCon hGH-treated children, compared to 9.04 cm/year for those treated with daily somatropin. In the trial, TransCon hGH demonstrated a safety and tolerability profile similar to daily somatropin through follow-up of up to 143 weeks. Adverse events were mild to moderate in severity, with no AEs leading to discontinuation of study drug.

Ascendis Pharma "announced new data from its pivotal ApproaCH Trial showing that children with achondroplasia treated with once-weekly TransCon CNP (navepegritide) maintained consistent improvements in growth through Week 104, with further improvement in body proportionality during the second year of treatment. These and other results were shared in an oral presentation by Dr. Ravi Savarirayan, M.B.B.S., M.D., Murdoch Children's Research Institute (Australia), during ACMG 2026, the Annual Clinical Genetics Meeting held March 10-14 in Baltimore, Maryland. TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. In February 2026, TransCon CNP was approved by the U.S. FDA under the trade name YUVIWEL(R) to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. The Marketing Authorization Application for YUVIWEL is under review by the European Medicines Agency with a regulatory decision anticipated in the fourth quarter of 2026."