Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies.

ABSTRACT:

Background

Achondroplasia is the most common short-limbed skeletal dysplasia resulting from gain-of-function pathogenic variants in fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Most treatment options are symptomatic, targeting medical complications. Infigratinib is an orally bioavailable, FGFR1-3 selective tyrosine kinase inhibitor being investigated as a direct therapeutic strategy to counteract FGFR3 overactivity in achondroplasia.

Objectives

The main objective of PROPEL is to collect baseline data of children with achondroplasia being considered for future enrollment in interventional studies sponsored by QED Therapeutics. The objectives of PROPEL 2 are to obtain preliminary evidence of safety and efficacy o

SUBMITTER: Savarirayan R 

PROVIDER: S-EPMC8941703 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature