hrp0095p1-307 | Growth and Syndromes | ESPE2022

A Prospective Clinical Trial of Vosoritide in Hypochondroplasia: Baseline Demographics and Preliminary Results

Dauber Andrew , McCarthy Tara , Zhang Anqing , Merchant Nadia , Boucher Kimberly , Dham Niti , Kanakatti Shankar Roopa

Objectives: Vosoritide is a C-type natriuretic peptide analog which binds its receptor on chondrocytes, leading to increased chondrocyte proliferation and differentiation via its inhibition of the ERK1/2-MAPK pathway. It was recently approved for increasing linear growth in children with achondroplasia. This is the first study to examine the safety and efficacy of vosoritide in children with hypochondroplasia.Methods: Th...

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...