patidegib

Patidegib is a small molecule, cyclopamine-derived inhibitor of the Hedgehog (Hh) signaling pathway. It acts by binding to and inhibiting the G-protein coupled receptor smoothened (SMO), thereby blocking Hh pathway signaling, which leads to decreased proliferation and survival of tumor cells with overactive Hh pathways[1][4][5]. Patidegib has been developed in both oral and topical gel formulations. The drug is primarily being investigated for the prevention and treatment of basal cell carcinomas (BCCs), particularly in patients with Gorlin syndrome, a rare genetic disorder characterized by multiple BCCs due to PTCH1 mutations[3][6][7]. Patidegib has also been studied for conventional chondrosarcoma[2][5]. It has received Orphan Drug Designation and Breakthrough Therapy Designation from regulatory agencies for its use in Gorlin syndrome-related BCCs[7][8].

Brand names

SGT-610SGT610SGT 610

Other names

saridegibpatidegib topical gel