Patidegib topical gel
Topical HH Inhibitor (Patidegib) for Gorlin Syndrome
Gorlin syndrome, Frequent basal cell carcinoma
PTCH1 (patched-1, a smoothened inhibitor)
topical small molecule
Patidegib topical gel is designed to selectively replace and/or augment the function of Patched (Ptc), a naturally occurring hedgehog signaling pathway inhibitor, by blocking the Smoothened (Smo) protein. Acting as a tumor suppressor, patidegib is designed to allow cells to function normally and limit the overproduction of tumors found in patients with Gorlin Syndrome and High Frequency Basal Cell Carcinoma. Because oral formulations for this treatment typically have serious systemic side effects which cause many patients to discontinue treatment, patidegib is formulated as a topical gel to reduce its side effect profile due to its minimal systemic exposure. BridgeBio is currently investigating the safety and efficacy of patidegib in patients with Gorlin Syndrome in a Phase 3 clinical trial.