Topical patidegib granted FDA breakthrough, orphan statuses for Gorlin syndrome

The FDA has granted breakthrough therapy designation and orphan drug designation to topical patidegib for the treatment of basal cell carcinoma nevus syndrome, which is also known as Gorlin syndrome, according to a press release from PellePharm.

The approval is so the drug may be used to reduce morbidity and disease burden for patients who develop basal cell carcinomas (BCCs) from this syndrome.

The approval came in the wake of phase 2 data that showed the drug demonstrated safety and efficacy within the hedgehog-signaling pathway when administered topically, according to the release. It was also noted the gel formulation may be stably kept at room temperature for at least 2 years, allowing patients to manage their own disease. Phase 3 studies will begin in 2018.

The orphan drug designation was granted because the syndrome has a comparatively low prevalence in the general population.

“These two significant FDA designations mark important steps forward in our development of topical patidegib as a drug with substantial potential to reduce the BCC tumor burden in Gorlin syndrome patients,” Ervin Epstein, MD, co-founder and chief medical officer of PellePharm, said in the release. He added, “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.”

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