Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia.

ABSTRACT:

Background and objective

Vosoritide, an analog of C-type natriuretic peptide, has been developed for the treatment of children with achondroplasia. The pharmacokinetics of vosoritide and relationships between plasma exposure and efficacy, biomarkers, and safety endpoints were evaluated in a phase II, open-label, dose-escalation study (N = 35 patients aged 5-14 years who received daily subcutaneous injections for 24 months) and a phase III, double-blind, placebo-controlled study (N = 60 patients aged 5-18 years randomized to receive daily subcutaneous injections for 52 weeks).

Methods

Pharmacokinetic parameters for both studies were obtained from non-compartmental analysis. Potential correlations between vosoritide exposure and changes in annualized growth velocity, collagen

SUBMITTER: Chan ML 

PROVIDER: S-EPMC8813707 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature