PHILADELPHIA, PA — Medicus Pharma Ltd. (Nasdaq: MDCX) has asked the U.S. Food and Drug Administration to grant Rare Pediatric Disease Designation for its experimental SkinJect therapy in patients with Gorlin syndrome, a rare genetic disorder that can cause hundreds of recurring basal cell carcinomas over a lifetime.
The designation request marks another step in the company’s regulatory strategy for SkinJect, an investigational microneedle patch designed to deliver the chemotherapy drug doxorubicin directly into skin cancer lesions. The application follows a previously submitted orphan drug designation request and a proposed registrational study that remains under FDA review.
Gorlin syndrome, also known as Nevoid Basal Cell Carcinoma Syndrome, is a rare inherited disorder affecting an estimated 6,000 to 12,000 people in the United States. Patients often develop basal cell carcinomas beginning in childhood or adolescence and may require repeated surgeries throughout their lives.
Medicus is positioning SkinJect as a potential non-surgical alternative for treating those lesions.
The company’s recently completed Phase 2 trial reported clinical clearance in 64% of evaluable patients treated with the highest dose of the therapy, while 55% achieved complete histological response. The study also reported a favorable safety and tolerability profile.
SkinJect uses a dissolvable microneedle array to deliver doxorubicin directly into nodular basal cell carcinoma lesions. The localized approach is intended to concentrate treatment at the tumor site while limiting systemic exposure to the drug.
“Patients with Gorlin Syndrome often face a lifetime of recurring surgeries and progressive treatment burden beginning at a young age,” said Dr. Raza Bokhari, Medicus’ executive chairman and chief executive officer. He said the company believes SkinJect could become an important treatment option for patients seeking alternatives to repeated surgical procedures.
If granted, Rare Pediatric Disease Designation could make SkinJect eligible for a Priority Review Voucher should the therapy ultimately receive FDA approval for the designated indication.
Such vouchers can be used to obtain an expedited FDA review of another drug application or sold to another company. Priority review typically shortens FDA review timelines from approximately 10 months to six months.
The company noted that a separate orphan drug designation, if approved and followed by a successful marketing application, could provide seven years of market exclusivity in the United States, along with fee waivers and other development incentives.
Medicus said it plans to continue discussions with regulators regarding the clinical development pathway for SkinJect in Gorlin syndrome while pursuing broader applications in basal cell carcinoma treatment.
The company’s lead development program targets a condition for which no FDA-approved therapies currently exist for pediatric patients and where treatment often relies on repeated surgical intervention.
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