Ascendis Pharma A/S is scheduled to present two-year data from its ApproaCH trial of TransCon CNP at the International Skeletal Dysplasia Society (ISDS) annual meeting in Toronto this week.
The presentation, scheduled for Friday at 10:00 a.m. during Session 4, will cover Week 104 results from the open-label extension of the trial in children with achondroplasia. The data will be delivered by Carlos Bacino, M.D., Professor of Molecular and Human Genetics at Baylor College of Medicine and Texas Children’s Hospital. Separately, Ascendis will also present a poster analyzing injection site reactions in cases where indirect treatment comparison is not feasible.
TransCon CNP, marketed as YUVIWEL, is a prodrug of C-type natriuretic peptide designed for once-weekly administration. The therapy aims to provide continuous exposure of active CNP to receptors, counteracting overactive FGFR3 signaling associated with achondroplasia.
Achondroplasia, a rare genetic condition caused by a fibroblast growth factor receptor 3 (FGFR3) variant, affects more than 250,000 people worldwide. The condition is linked to spinal abnormalities, enlarged brain ventricles, impaired muscle strength, hearing deficits, upper airway obstructions, and chronic pain.
Ascendis received U.S. FDA approval for TransCon CNP in February 2026 to increase linear growth in pediatric patients aged two years and older with achondroplasia and open epiphyses. The company’s Marketing Authorisation Application with the European Medicines Agency remains under review, with a regulatory decision expected in the fourth quarter of 2026.
