Medicus Pharma files Orphan Drug application for SkinJect in rare skin Cancer indication

Medicus Pharma CEO Dr Raza Bokhari joined Steve Darling from Proactive to announce that the company has submitted an Orphan Drug Designation application to the U.S. Food and Drug Administration for SkinJect® as a treatment for basal cell carcinoma in patients with Gorlin Syndrome. Bokhari explained that the submission represents a strategic expansion of the SkinJect program into a high unmet-need orphan indication, where current treatment options are limited and often require repeated surgical procedures that can lead to cumulative morbidity and disfigurement. Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare inherited disorder typically caused by mutations in the PTCH1 gene. This leads to dysregulation of the Hedgehog signaling pathway and the development of multiple, recurring skin cancers. Patients may develop dozens to more than 1,000 basal cell carcinomas over their lifetime, often beginning in childhood, creating a significant physical and psych...