hrp0094p2-303 | Growth and syndromes (to include Turner syndrome) | ESPE2021

A literature review of the potency and selectivity of FGFR-selective tyrosine kinase inhibitors, such as infigratinib, in the potential treatment of achondroplasia

Dobscha Katherine , Wei Ge , Dambkowski Carl , Rogoff Daniela ,

Background: Germline mutations in fibroblast growth factor receptor (FGFR) genes 1–3 can cause skeletal dysplasias such as achondroplasia (ACH), which is caused primarily by a G380R substitution in FGFR3. Infigratinib (BGJ398), a potent and selective FGFR1–3 tyrosine kinase inhibitor (TKI), demonstrated preclinical efficacy at low doses in an ACH mouse model. The objective of this analysis is to evaluate dose dependency and toxicity...