FRAX486 is a potent and selective inhibitor of p21-activated kinase (PAK), particularly targeting group I PAKs (PAK1-3). It is primarily used in research settings to study signaling pathways involved in cell proliferation, cytoskeletal dynamics, and neuronal function. Due to its ability to cross the blood-brain barrier, FRAX486 has been investigated in preclinical models of neurological disorders, including fragile X syndrome and other autism spectrum disorders, where aberrant PAK signaling is implicated. It has shown potential in reversing synaptic and behavioral deficits in animal models, suggesting possible therapeutic relevance for neurodevelopmental conditions. Additionally, FRAX486 is used in cancer research to explore the role of PAK in tumor growth, invasion, and metastasis, particularly in breast cancer and glioblastoma models. Its application remains experimental, mainly confined to in vitro and in vivo research.