• Alias: GDC-0449
    • An orally bioavailable small molecule that targets the Hedgehog (Hh) signaling pathway, blocking the activities of the Hh-ligand cell surface receptors patched and/or SMO and suppressing Hh signaling
    • FDA approved for basal cell carcinoma
    • First FDA-approved Hh signaling agent
    • Under clinical investigation for other cancers including colorectal cancer, NSCLC, and pancreatic cancer
    • Recommended dose: 150 mg PO once daily
    • Half-life: 12 days after single dose, 4 days after continuous once-daily dosing
    • Metabolism: Weak CYP2C9 and CYP3A4 substrates
    • Common side effects: Embryo–fetal death and severe birth defects (US box warning), pasms, alopecia, dysgeusia, weight loss, fatigue, nausea, diarrhea
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