http://www.raredr.com/news/fda-breakthrough-therapy-designation-gorlin-syndrome-treatment
FDA Grants Breakthrough Therapy Designation to PellePharm for Gorlin Syndrome Treatment

Mathew Shanley

This morning, the U.S. Food and Drug Administration (FDA) granted both Breakthrough Therapy Designation and Orphan Drug Designation to PellePharm for its compound topical patidegib. The therapy is intended for use in patients with Basal-cell nevus syndrome (BCNNS).

Topical patidegib is designed to reduce the life-long serious clinical morbidity and disease burden of persistently developing basal cell carcinomas in this patient population.

Basal-cell nevus syndrome, commonly referred to as Gorlin Syndrome, is a genetic condition that is characterized by defects within multiple body systems, like the skin, nervous system, eyes, endocrine system, and bones.

The Breakthrough Therapy Designation was granted based on results from PellePharm’s Phase 2 trial evaluating the safety and efficacy of topical patidegib in patients with BCNNS. Topline data from the study were published in July 2017, and showed that the drug was successful in blocking the disease at its source within the hedgehog pathway. This effect was previously demonstrated by oral patidegib in Phase 1 trials, but the results of the Phase 2 was the first instance of zero adverse systemic side effects of oral hedgehog inhibitors.

The 10,000 patients with a confirmed Gorlin syndrome diagnosis led to the additional Orphan Drug Designation.

“Our drug is a hedgehog inhibitor, and hedgehog inhibitors can make the huge difference in the lives of patients with basal cell carcinomas, even if they have too many to count, like the group we’re interested in,” said Ervin Epstein, Co-Founder and Chief Medical Officer for PellePharm in an exclusive interview with Rare Disease Report. “The tumors can go away completely, as we and others have found, but they can recur if patients stop taking the drug because of uncomfortable side effects, like hair loss.”

At present, there are no approved therapies for BCNNS, and the current standard of care for the disease is surgery. Patients with the severe cases can have up to 30 surgeries per year.

“Results of our Phase 2 were good enough to the point that we were enthused to continue the development of the drug, that will hopefully lead us to a Phase 3 trial,” said Epstein.

“We’re of course, really excited about the organizational commitment from the FDA to develop the drug as quickly and efficiently as possible,” added Alix Alderman, Vice President of Regulatory Affairs at PellePharm.

The timing of PellePharm’s follow-up meeting with the FDA has not yet been agreed upon, however, it can be assumed that the next steps in the process for the company and topical patidegib will be put into motion in early 2018.

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