Model Low-dose Infigratinib -FGFR1-3 Inhibitor for Achondroplasia

Infigratinib (BGJ398), is an orally administered, ATP-competitive, FGFR1-3 tyrosine kinase inhibitor in development for the treatment of FGFR-driven conditions, including achondroplasia, a bone growth disorder in children. Overactivating FGFR3 mutations drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing disproportionate short stature and serious health complications stemming from cranial and spinal defects. Low dose infigratinib has best-in-class potential due to its design to inhibit mutant FGFR3 receptor. In mouse models of achondroplasia, infigratinib demonstrated robust bone growth and clear improvement in cranial and spinal defects. BridgeBio is currently conducting a Phase 2 clinical trial in achondroplasia patients to evaluate the safety and tolerability of low-dose infigratinib, as well as clinical proof-of-concept.