hrp0098fc5.5 | Growth and Syndromes | ESPE2024

Phase 2 Trial of Vosoritide Use in patients with Hypochondroplasia: Pharmacokinetic/ Pharmacodynamic analysis from 12 Month Data

Galetaki Despoina , Zhang Anqing , Qi Yulan , merchant Nadia , Kanakatti Shankar Roopa , Boucher Kimberly , Shafaei Niusha , Seaforth Raheem , Dham Niti , Dauber Andrew

Objectives: Vosoritide is a C-type natriuretic peptide (CNP) analog that binds its receptor on chondrocytes, promoting growth by inhibiting the ERK1/2-MAPK pathway. We previously reported the results of a Phase II study in children with hypochondroplasia. Vosoritide led to an average increase in annualized growth velocity (GV) of 1.81 cm/year and gain of 0.36 in height SD over 12 months. We present here the pharmacokinetic/ pharmacodynamic (PK/PD) data from th...