PellePharm, a clinical-stage biopharmaceutical company focusing on rare genetic dermatological conditions, announced that the 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 Nevus Syndrome (BCNNS), a rare genetic disease also known as Gorlin Syndrome.
“These two significant FDA designations mark important steps forward in our development of topical patidegib as a drug with a substantial potential to mitigate the BCC tumor burden in Gorlin Syndrome patients. From our co-discovery of the underlying genetic defect, through over two decades of preclinical research with hedgehog inhibitors, and our recent clinical trials, our team at PellePharm is deeply committed to delivering a safe and effective therapy for people living with Gorlin Syndrome,” said Ervin Epstein, MD, cofounder and chief medical officer of PellePharm. “We are eager to continue our development program and work closely with the FDA and other regulatory authorities to deliver this potential first-in-class therapy to patients as quickly as possible.”
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.
“We are pleased that the FDA recognizes the importance of accelerating the development of topical patidegib for people affected by BCCNS (Gorlin Syndrome),” said Jean Pickford, executive director of the Basal Cell Carcinoma Nevus Syndrome Life Support Network. “People living with BCCNS need additional therapeutic options to help lessen the burden of disease.”
Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery, with patients with severe disease having as many as 30 surgeries per year.
PellePharm intends to initiate a Phase 3 clinical trial for patidegib in Gorlin Syndrome in 2018. Topical patidegib gel has shown early promise in a Phase 2 clinical study in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials, but without the adverse systemic side effects of oral hedgehog inhibitors. Patidegib’s gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome.
Topical patidegib currently is being studied in a United States-based Phase 2 clinical trial for the treatment of sporadic BCCs.
