Ascendis Pharma Presents Two-Year ApproaCH Trial Data at ISDS 2026, Showing Durable Growth and Alignment Gains for FDA-Approved YUVIWEL

Ascendis Pharma A/S announced on August 25, 2026 that it will deliver an oral presentation of Week 104 data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) at the International Skeletal Dysplasia Society annual meeting in Toronto, running August 26–29, 2026. The presenter is Carlos Bacino, M.D., FACMG, Professor of Molecular and Human Genetics at Baylor College of Medicine and Texas Children's Hospital.

The ApproaCH Trial enrolled 84 children aged 2–11 years with achondroplasia in a randomized, double-blind, placebo-controlled design. Participants received TransCon CNP at 100 µg/kg once weekly or placebo for 52 weeks, then all rolled into an open-label extension in which every participant received TransCon CNP through Week 104.

The two-year readout shows continued improvements in lower extremity alignment, including tibial-femoral angle, through up to two years of treatment. Improvements in annualized growth velocity were maintained, and achondroplasia-specific height Z-score increased with TransCon CNP treatment through Week 104, per the company's August 25, 2026 news release on GlobeNewswire.

The U.S. Food and Drug Administration approved TransCon CNP under the trade name YUVIWEL® in February 2026 to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. A Marketing Authorisation Application for YUVIWEL is currently under review by the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026, per the company's August 25, 2026 news release.

On mechanism: TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous CNP exposure to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling that underlies achondroplasia. The condition — a rare genetic disorder arising from a systemic FGFR3 variant that disrupts the balance between FGFR3 and CNP signaling pathways — is estimated to affect more than 250,000 people worldwide, per the company's August 25, 2026 release.

On the balance sheet: as of September 30, 2025, Ascendis Pharma reported cash and cash equivalents of €539 million, per SEC filing. The company reported Q2 2026 earnings on August 13, 2026, though full financial details from that report were not accessible in available sources. Ascendis carries 62,376,846 shares outstanding and a market cap of $15.21 billion, per TipRanks.

No executive quote was included in the ISDS presentation announcement itself.

What's next: the EMA regulatory decision on YUVIWEL is expected in Q4 2026, per the company's August 25, 2026 news release.