BRIEF—Breakthrough and Orphan designations for PellePharm's topical patidegib in Gorlin syndrome

California, USA-based PellePharm says that the US Food and Drug Administration has granted both Breakthrough Therapy and Orphan Drug designations to topical patidegib for reduction of the life-long serious clinical morbidity and disease burden of persistently developing basal cell carcinomas (BCCs) in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), a rare genetic disease also known as Gorlin syndrome.

Currently, there are no FDA-approved therapies for Gorlin syndrome, and the standard of care for this rare disease is surgery, according to PellePharm. Patients with severe disease have as many as 30 surgeries per year.

PellePharm intends to initiate a Phase III clinical trial for patidegib in Gorlin syndrome in 2018.

The FDA granted PellePharm Breakthrough Therapy designation based on results from its Phase II trial assessing the safety and efficacy of topical patidegib in patients with Gorlin syndrome. Top-line data from this study were announced in July 2017.

Breakthrough Therapy designation is designed to expedite the development and review of drugs that are intended to treat a serious condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.