ESPE Abstracts

Background: Achondroplasia (ACH), the most common short-limbed skeletal dysplasia, is characterized by impaired endochondral ossification resulting from gain-of-function pathogenic variants in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone growth. People with ACH are at risk for several significant co-morbidities, including brainstem compression due to foramen magnum stenosis, sleep-disordered breathing, chronic...

Objectives: Achondroplasia (ACH) is characterized by disproportionate short stature and is associated with medical complications and functional constraints. Infigratinib is an oral FGFR3 inhibitor that targets overactive FGFR3 signaling at its origin and is under development as a treatment option for children with ACH. We report Month 18 results from PROPEL 2 (NCT04265651), a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluat...