Vismodegib (GDC-0449) is a potent, selective, orally bioavailable smoothened (Smo) inhibitor, blocks Shh signaling with EC50 of 40 nM.
Vismodegib (GDC-0449) inhibits proliferation, increases apoptosis, and alters morphology, suppresses the Shh pathway in mouse medulloblastoma in vivo.
Vismodegib (GDC-0449) also is a potent inhibitor of ABCG2/BCRP and ABCB1/Pgp.
Vismodegib (GDC-0449) is the first Hedgehog signaling pathway targeting agent to gain FDA approval.
