We currently see many unmet needs within BCCNS as currently the standard of care is surgery which typically results in facial disfigurement. The current approved hedgehog inhibitors have intolerable toxicity, so they are reserved for irresectable lesions. Hedgehog signaling must be suppressed on a continual basis to inhibit new tumor development and growth of existing tumors. We believe that itraconazole is the answer to the need for a chronic, well-tolerated therapy that results in tumor control minimizing surgical procedures otherwise required.
The Gorlin Syndrome Alliance’s Voice of the Patient Report, an Externally Led, Patient Focused, Drug Development Meeting sums up why there is a drastic unmet need in BCCNS.
