Patidegib Gets Breakthrough and Orphan Designation for Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by mutations in the tumor suppressor gene encoding Patched1.

The Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation and Orphan Drug Designation to patidegib (PellePharm). This topical treatment is being investigated for the reduction of life-long serious clinical morbidity and disease burden of persistently developing basal cell carcinomas in patients with basal cell carcinoma nevus syndrome, also known as Gorlin Syndrome.

Topical patidegib gel has already shown promise in a Phase 2 study; the drug works by blocking the disease at its source within the hedgehog signaling pathway. Unlike oral hedgehog inhibitors, the gel carries no adverse systemic effects; it is also stable at room temperature for at least 2 years making it a viable option for ongoing, at-home treatment. 

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Currently, there are no FDA-approved treatments for Gorlin Syndrome. The company plans on starting a Phase 3 clinical trial in 2018.

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