FDA grants Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for topical patidegib in Gorlin syndrome.
PellePharm, a clinical-stage biopharmaceutical company targeting rare genetic dermatological conditions at the source, announced that the U.S. Food and Drug Administration (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 (BCCs) in patients with basal cell carcinoma nevus syndrome (BCCNS), 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. 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.
The FDA granted Orphan Drug Designation based on the relatively low prevalence of Gorlin syndrome. The FDA’s Orphan Drug Designation program is designed to incentivize and facilitate the development of drugs for rare diseases that affect fewer than 200,000 people in the U.S. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin syndrome.
Currently, there are no FDA-approved therapies for Gorlin syndrome, and the standard of care for this rare disease is surgery. Patients with severe disease have as many as 30 surgeries per year.
PellePharm intends to initiate a Phase 3 clinical trial for patidegib in Gorlin syndrome in 2018.
(Source: PellePharm, Inc.)
Filed Under: Drug Discovery