Posted by Michael Wonder on 21 Nov 2017
FDA grants breakthrough therapy designation and orphan drug designation to PellePharm for topical patidegib in Gorlin syndrome
20 November 2017 - PellePharm today announced that the U.S. FDA has granted both breakthrough therapy designation and orphan drug designation to topical patidegib for reduction of the life-long serious clinical morbidity and disease burden of persistently developing basal cell carcinomas in patients with basal cell carcinoma nevus syndrome, a rare genetic disease also known as Gorlin syndrome.
The FDA granted PellePharm breakthrough therapy designation based on results from its Phase 2 trial assessing the safety and efficacy of topical patidegib in patients with Gorlin syndrome. Topline data from this study were announced in July 2017.
