Patidegib has orphan drug and breakthrough therapy designations from the FDA and an orphan drug designation from the European Medicines Agency as a topical agent for Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, a rare genetic form of BCC characterized by mutations in PTCH1 resulting in multiple BCCs [20, 21]. The gene discussed is PTCH1; the disease is nevoid basal cell carcinoma syndrome.