Infigratinib for the treatment of FGFR3-related bone disease during pregnancy

摘要

Acquired mutations in FGFR3 are a family of cartilage dysplasias, namely achondroplasia (ACH), the most common form of short stature, and tanatophoric, the fatal form of short stature. It is involved in osteodysplasia (TD) and achondroplasia (HCH). Recent data support that infigratinib (NVP-BGJ398) corrects the pathological features of achondroplasia and supports infigratinib as a potential therapeutic approach for FGFR3-related bone disease. Here we investigate the feasibility of using drugs to treat skeletal incomplete growth during pregnancy. We treated pregnant female Fgfr3 Neo / Y367C mice with a drug (4 mg / kg) subcutaneously injected daily from E14.5 day embryos until day 1 (postnatal). The data showed that treatment with BGJ398 for 5 days in pregnant mice successfully suppressed 15 skeletal abnormalities that occurred during the embryonic stage. Therefore, the present invention relates to a method for treating FGFR3-related bone disease during pregnancy using infigratinib.