ISSN 1662-4009 (online)

ey0021.5-12 | Novel Treatments | ESPEYB21

5.12. Once-weekly transCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-Blind, placebo-controlled, dose-escalation trial

Savarirayan Ravi , Hoernschemeyer Daniel G. , Ljungberg Merete , Zarate Yuri A. , Bacino Carlos A. , Bober Michael B. , Legare Janet M. , et al.

In brief: This multinational, randomised, double-blind, placebo-controlled, dose-escalation Phase 2 study evaluated the safety and efficacy of TransCon CNP (navepegritide), a C-type natriuretic peptide (CNP) analogue designed to allow continuous CNP exposure with once-weekly dosing, in 42 children with achondroplasia aged 2-10 years. The study was sponsored by Ascendis Pharma.Commentary: Achondroplasia is one of the most common constitutional bone disord...