Medicus Pharma (NASDAQ:MDCX) said Monday it has submitted a Rare Pediatric Disease Designation request to the US Food and Drug Administration for SkinJect, its investigational microneedle patch for treating basal cell carcinoma in patients with Gorlin Syndrome.
The submission follows an Orphan Drug Designation application and a registrational study design currently under FDA review for SkinJect, a dissolvable patch that delivers the chemotherapy drug doxorubicin directly into skin cancer lesions.
Gorlin Syndrome is a rare genetic disorder in which patients can develop dozens to more than a thousand basal cell carcinomas over a lifetime, with lesions often appearing in childhood. The condition affects an estimated 1 in 30,000 to 60,000 people worldwide, or roughly 6,000 to 12,000 patients in the US.
In a recently completed Phase 2 study, the 200-microgram SkinJect treatment arm showed a 64% clinical clearance rate and a 55% complete response rate among evaluable nodular basal cell carcinoma patients.
"Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age," CEO Raza Bokhari said in a statement.
“We believe (SkinJect) has the potential to become an important non-surgical treatment option for these patients.”
If granted, the designation could make SkinJect eligible for a Rare Pediatric Disease Priority Review Voucher upon eventual FDA approval, which can be used to accelerate review of a future drug application.
A separate Orphan Drug Designation, if granted, could provide seven years of US market exclusivity and waivers of certain FDA application fees.